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Stowers scientists uncover a critical component that helps killifish regenerate their fins

The findings are a step toward closing the gap on how we could one day deploy regenerative medicine in humans

Stowers Institute for Medical Research

 

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Stowers scientists discuss findings of killifish limb regeneration research, shedding light on the evolutionary aspects of regeneration and the potential for developing novel therapeutic strategies in regenerative medicine for humans. 

 

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Credit: Stowers Institute for Medical Research

KANSAS CITY, MO—September 26, 2024—Spontaneous injuries like the loss of a limb or damage to the spinal cord are impossible for humans to repair. Yet, some animals have an extraordinary capacity to regenerate after injury, a response that requires a precise sequence of cellular events. Now, new research from the Stowers Institute for Medical Research has unveiled a critical timing factor—specifically how long cells actively respond to injury—involved in regulating regeneration.

A recent study published in iScience on September 20, 2024, sought to understand exactly how an organism knows how much tissue has been lost post-injury. Led by former Predoctoral Researcher Augusto Ortega Granillo, Ph.D., in the lab of Stowers President and Chief Scientific Officer Alejandro Sánchez Alvarado, Ph.D., the team investigated how African killifish properly regrow their tail fin following damage. By analyzing tissue dynamics during regrowth, they found that in addition to known factors, including how many cells are participating and where they are located, the length of time cells spend engaged in the repair process is also key.

“One of the greatest unsolved mysteries of regeneration is how an organism knows what has been lost after injury,” said Sánchez Alvarado. “Essentially, the study points to a new variable in the equation of regeneration. If we can modulate the rate and the length of time that a tissue can launch a regenerative response, this could help us devise therapies that may activate and perhaps prolong the regenerative response of tissues that normally would not do so.”

Shortly after a killifish tail injury, the remaining tissue needs to know how much damage has occurred. Then, this tissue must enlist the right number of repair cells to the site of injury for the right amount of time. Damage sensing, repair cell recruitment, and timing somehow must work together to regrow the tail.

“If an animal that can regenerate extremities, like a tail, loses just a tiny portion, how does it know not to regenerate a whole new tail but just the missing piece?” said Sánchez Alvarado. To address this question, the team probed different locations of injury in the killifish tail fin.

They found that skin cells both near an injury and in distant, uninjured regions launch a genetic program that primes the whole animal to prepare for a repair response. Then, skin cells at the site of injury sustain this response and temporarily change their state to modify the surrounding material called the extracellular matrix. Ortega Granillo likens this matrix to a sponge that absorbs secreted signals from the injured tissue that then guides repair cells to get to work. If the signals are not received or not interpreted correctly, the regeneration process may not restore the tail’s original shape and size.

“We very clearly defined when and where—at 24 hours post-injury and in the extracellular matrix—the transient cell state is acting in the fin tissue,” said Ortega Granillo. “Knowing when and where to look allowed us to make genetic disruptions and gain a better understanding of the function of these cell states during regeneration.”

To investigate whether these distinct cellular states communicate information to the extracellular matrix—the supportive structure surrounding cells—during the repair process, the researchers employed the CRISPR-Cas9 gene editing technique. They specifically targeted a gene known to modify the extracellular matrix, as they had observed its activation at the onset of the regeneration response. By disrupting the function of this gene, the team aimed to determine its role in relaying information from cells to the matrix during regeneration.  

“These modified animals no longer know how much tissue was lost,” said Ortega Granillo. “They still regenerated, but the speed of tissue growth was deficient. This is telling us that by changing the extracellular space, skin cells inform the tissue how much was lost and how fast it should grow.”

Indeed, the speed and amount of tissue regenerated in these genetically modified killifish increased regardless of whether the tail injury was mild or severe. This finding opens the possibility that cell states that modify the matrix increase regenerative regrowth. If the cell states could be adjusted, it may be a way to stimulate a more robust regeneration response.  

From an evolutionary perspective, understanding why certain organisms excel at regeneration while others, such as humans, have limited regenerative abilities is a driving force in the field of regenerative biology. By identifying general principles in organisms with high regenerative capacity, researchers aim to potentially apply these insights to enhance regeneration in humans. This comparative approach not only sheds light on the evolutionary aspects of regeneration but also holds promise for developing novel therapeutic strategies in regenerative medicine.

“Our goal is to understand how to shape and grow tissues,” said Ortega Granillo. “For people who sustain injuries or organ failure, regenerative therapies could restore function that was compromised during illness or following injury.”

Additional authors include Daniel Zamora, Robert Schnittker, Allison Scott, Alessia Spluga, Jonathon Russell, Carolyn Brewster, Eric Ross, Daniel Acheampong, Ning Zhang, Ph.D., Kevin Ferro, Ph.D., Jason Morrison, Boris Rubinstein, Ph.D., Anoja Perera, and Wei Wang, Ph.D.

This work was funded by institutional support from the Stowers Institute for Medical Research and the Howard Hughes Medical Institute.

About the Stowers Institute for Medical Research

Founded in 1994 through the generosity of Jim Stowers, founder of American Century Investments, and his wife, Virginia, the Stowers Institute for Medical Research is a non-profit, biomedical research organization with a focus on foundational research. Its mission is to expand our understanding of the secrets of life and improve life’s quality through innovative approaches to the causes, treatment, and prevention of diseases.

The Institute consists of 21 independent research programs. Of the approximately 500 members, over 370 are scientific staff that include principal investigators, technology center directors, postdoctoral scientists, graduate students, and technical support staff. Learn more about the Institute at www.stowers.org and about its graduate program at www.stowers.org/gradschool.

The African Killifish 

Shortly after a killifish tail injury, the remaining tissue needs to know how much damage has occurred. Then, this tissue must enlist the right number of repair cells to the site of injury for the right amount of time. Damage sensing, repair cell recruitment, and timing somehow must work together to regrow the tail.

Credit

Stowers Institute for Medical Research

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Journal

iScience

Method of Research

Experimental study

Subject of Research

Animals

Article Title

Positional information modulates transient regeneration-activated cell states during vertebrate appendage regeneration

Article Publication Date

20-Sep-2024

 

CRISPR/Cas9 modifies euglena to create potential biofuel source

Mutant microalgae produce wax esters for biofuel feedstock with improved cold flow

Osaka Metropolitan University

 

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Genome editing of Euglena gracilis led to stable mutants that produced wax esters with shorter carbon chains, giving the esters properties more suitable for use in biofuel.

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Credit: Osaka Metropolitan University

News about biofuels sometimes mentions used cooking oil as a feedstock, but if these substances contain animal fat, they can solidify in colder temperatures. This happens because, chemically, the fatty acids of these and many other saturated fats have long carbon chains with single bonds. Enter the euglena. An Osaka Metropolitan University team has found a way to have one species of this microalgae produce wax esters with shorter carbon chains than usual.

Using CRISPR/Cas9 to edit the genome of Euglena gracilis, Dr. Masami Nakazawa and her team at the Graduate School of Agriculture’s Department of Applied Biochemistry produced stable mutants that created wax esters two carbons shorter than the wild-type species.

This improvement in the cold flow of the wax esters makes them more applicable as feedstock for biofuels. Among the factors favorable to using Euglena gracilis as a biofuel source are its ability to grow easily through photosynthesis and anaerobic production of wax esters.

“This achievement is expected to serve as a fundamental technology for replacing some petroleum-based production of wax esters with biological sources,” Dr. Nakazawa affirmed.

The findings were published in Bioresource Technology.

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About OMU 

Established in Osaka as one of the largest public universities in Japan, Osaka Metropolitan University is committed to shaping the future of society through “Convergence of Knowledge” and the promotion of world-class research. For more research news, visit https://www.omu.ac.jp/en/ and follow us on social media: X, Facebook, Instagram, LinkedIn.

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Journal

Bioresource Technology

DOI

10.1016/j.biortech.2024.131255 

Method of Research

Experimental study

Subject of Research

Cells

Article Title

Genome editing-based mutagenesis stably modif

 Research shows brain synchronization between humans and dogs

By Pooja Toshniwal Paharia

Reviewed by Danielle Ellis, B.Sc.

Sep 16 2024

Study reveals how mutual gazing and petting synchronize human and dog brains, while autism-related gene mutations in dogs reduce this connection. 

Study: Disrupted Human–Dog Interbrain Neural Coupling in Autism-Associated Shank3 Mutant Dogs. Image Credit: sergey kolesnikov/Shutterstock.com

In a recent study published in Advanced Science, researchers studied cross-species interbrain connections between dogs and humans. They also investigated whether autism-related gene abnormalities in dogs impede social interaction between human-dog pairs.

Background

The human-dog connection has developed with time, with dogs tamed for their protective and hunting capacities. They have become valuable members of households, offering companionship and emotional support. Interspecies partnerships generate mutual benefit but seldom approach the extent of communication between humans and dogs. Dogs can read, comprehend, and react to various human emotions and linguistic signs via facial expressions, behaviors, and voice tones. However, the brain mechanisms underlying interspecies social communication remain unknown.

About the study

In the present study, researchers investigated the brain processes enabling human-dog communication. They explored the influence of autism-related gene alterations in dogs on social interactions between the two species.

Non-invasive wireless electroencephalograms (EEG) concurrently detected brain activity in beagles (research canines) and humans during social interactions. To validate the findings, researchers assessed interbrain correlations between different areas of the brain under three situations. The situations included no social interactions in separate spaces, with social interaction in one room and without social engagement in one room. Social interactions included petting and mutual gazing.

Researchers compared interbrain coupling during complete social interactions (mutual gaze + petting) to partial social interactions (mutual gaze or petting alone) to evaluate the synergistic effects of mutual gaze and petting on interbrain coupling. They also investigated brain activity associations between dogs and human participants from different trials and recorded the brain activities of the two species during social interactions for five days to evaluate the impact of social familiarity on interbrain neural coupling.

Subsequently, researchers conducted an additional five-day investigation to assess the durability or changes in interbrain interactions across prolonged periods. Linear regressions investigated the association between the duration of social interactions and interbrain activity. Generalized partial directed coherence (GPDC) algorithms assessed the directionality of interbrain activity coupling.

Researchers developed an autism spectrum disorder (ASD) model for dogs with SH3 and multiple ankyrin repeat domain 3 (Shank3) mutations using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-associated protein 9 (Cas9) genome editing.

Behavioral studies such as the three-chamber test and human-dog interaction experiments revealed autism-like symptoms in the mutants. Over five days, researchers explored the interbrain neuronal connection between mutant canines and humans. Theta/beta wave ratios (TBR) indicated attention problems in the mutants during the social interactions between humans and dogs.

Researchers also explored the effects of lysergic acid diethylamide (LSD), a psychedelic, on brain function. They delivered a single dosage of 7.5 μg/kg bodyweight of LSD intramuscularly and observed its effects after 24 hours.

Results

Petting and mutual gazing resulted in interbrain synchronization in the parietal and frontal areas of the brain during human-dog interactions, respectively. These brain areas are involved in joint attention. The interbrain association in these brain areas of dogs and humans caused by mutual gazing or stroking alone was much lower than that during combined social interactions, including petting and mutual gazing.

Over five days, the synchronization intensity increased as the human-dog dyad became more familiar. Linear regression analyses revealed a strong positive association between social contact time, interbrain activity correlations, and GPDC values. After a week of social contacts, logistic growth curve regressions revealed that interbrain correlation in the frontal and parietal areas had plateaued. 

Interbrain correlations between humans and dogs in various sessions were much lower than in the same interaction sessions. The findings demonstrate that reciprocal involvement between dogs and humans is vital for interbrain neural connections. During the human-dog social interactions, the human takes the lead, and the dog follows. The mutant canines displayed lower attention and eliminated interbrain connections. A single dosage of LSD corrected the problems.

Conclusions

The study found that interbrain neural synchronizations between family dogs and human beings are identical to those observed during human-human interactions. The frontoparietal network is essential for interbrain activity coordination and sensory information attention. Dogs with Shank3 mutations demonstrated poor brain circuitry and attention, comparable to those with ASD. A single dosage of LSD restored reduced interbrain connection and joint attention in the mutant dogs, indicating that LSD may improve social impairment in ASD patients.

 The findings point to possible interbrain neural activity biological markers for autism spectrum disorder diagnosis and the development of designed non-hallucinogenic LSD analogs to address social deficiencies. Further research into brain coupling may improve the knowledge of the neurological mechanisms that underpin social interactions between regularly developing humans and those with mental illnesses like ASD.

Journal reference:

• Wei Ren, Shan Yu, Kun Guo, Chunming Lu, and Yong Q. Zhang. Disrupted Human-Dog Interbrain Neural Coupling in Autism-Associated Shank3 Mutant Dogs, Adv. Sci. 2024, 2402493, DOI: https://doi.org/10.1002/advs.202402493 https://onlinelibrary.wiley.com/doi/10.1002/advs.202402493?utm_campaign=publicity

A Boy And His Dog (1975) Official Trailer

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Oluwatayo Ajayi-Moses Unveils Plans to Tackle Plant Disease That Threatens Food SecurityFacebookTwitterWhatsApp

FlipboardLinkedInRedditEmailShare10 September 2024

Vanguard (Lagos)

By Ayo Onikoyi

Oluwatayo Ajayi-Moses, a dedicated researcher in genomics, phenomics, and bioinformatics, is on a mission to tackle one of the most pressing challenges in agriculture today: plant diseases that threaten food security.

With over ten years of experience in microbiological techniques and currently pursuing a Ph.D. at North Dakota State University, Ajayi-Moses is leveraging cutting-edge scientific methods to safeguard crops and enhance food production.

Ajayi-Moses's passion lies in decoding the genetic mysteries of small grains to develop more resilient crops. Through his work, he aims to mitigate the effects of plant pathogens, ensuring a stable and secure food supply for the United States and beyond. His research focuses on identifying genetic markers that can help in breeding disease-resistant plants, ultimately reducing the need for chemical pesticides and fostering sustainable farming practices.

As a researcher specializing in plant molecular genetics and bioinformatics, my goal is to develop innovative solutions to combat plant diseases and enhance food security.

His impactful research involves utilizing advanced techniques such as genomic library preparation, molecular cloning, and CRISPR-Cas9 gene editing. These methods allow him to investigate and manipulate the genetic material of plants, aiming to enhance their resistance to diseases and environmental stressors. Ajayi-Moses has a proven track record of scientific excellence, with thirteen published scientific articles and presentations at international conferences, highlighting his contributions to the fields of plant genetics and agricultural biotechnology.

With extensive experience as a Graduate Research Assistant at the Small Grains Genotyping Laboratory, under the U.S. Department of Agriculture, Ajayi-Moses collaborates with multidisciplinary teams to conduct genomic analyses on small grains. His work is crucial in identifying genetic diversity and agronomic traits that can be harnessed to improve crop resilience and yield.

My specialties include genomic data analysis, high-throughput data analysis, and bioinformatics. I also have extensive experience in computational biology, Python programming, R Studio, and molecular diagnostics.

During his academic tenure, Ajayi-Moses has also demonstrated strong leadership skills, serving as President of the Genomics Phenomics and Bioinformatics Student Association at North Dakota State University. His commitment to advancing plant science research is evident in his proactive approach to mentoring students and his active participation in various academic societies.

Ajayi-Moses's ability to translate complex genomic data into practical agricultural solutions has positioned him as a leading figure in plant disease research. His dedication to enhancing food security through innovative genetic research aligns with the national interest, as it contributes to the sustainability of the agricultural sector and the well-being of the U.S. population.

With a robust background in microbiology, genomics, and bioinformatics, Ajayi-Moses is driving forward the frontier of plant science, making significant strides in the fight against plant diseases. His work not only has the potential to revolutionize crop protection but also to ensure a more resilient and secure food future for generations to come.

Read the original article on Vanguard.

Gene therapy restores vision in first-ever trial for rare, inherited blindness

Patients born with a certain genetic blindness experienced the world in an entirely new way after a single treatment with gene therapy developed at UF.

University of Florida

After the treatment, one patient saw her first star. Another saw snowflakes for the first time. Other patients were newly able to navigate outside of the home or to read the labels on their child’s Halloween candy.

The cause of these seemingly miraculous improvements? A gene therapy developed by University of Florida scientists, which restored useful vision to most patients with the rare, inherited blindness known as Leber congenital amaurosis type I, or LCA1, in a small trial. 

Those who received the highest dose of the gene therapy saw up to a 10,000-fold improvement in their light sensitivity, were able to read more lines on an eye chart, and improved in their ability to navigate a standardized maze. For many patients, it was akin to finally turning on dim lights after trying to navigate their homes in the pitch black for years, the researchers said.

The trial also tested the safety profile of the treatment. Side effects were largely limited to minor surgical complications. The gene therapy itself caused mild inflammation that was treated with steroids.

“This is the first time that anyone with LCA1 has ever been treated, and we showed a very clean safety profile, and we also showed efficacy. These results pave the way for advancing the therapy in a phase 3 clinical trial and eventually commercializing it,” said Shannon Boye, Ph.D., chief of the Division of Cellular and Molecular Therapy at UF, co-author of the study and co-founder of Atsena Therapeutics, the UF spinoff that developed the gene therapy and funded the study.

“Atsena is pleased to advance the foundational work that Shannon and Sanford Boye developed in their laboratory many years ago and thrilled that the 12-month data from our ongoing clinical trial have been published in a prestigious medical journal,” said Kenji Fujita, M.D., chief medical officer of Atsena Therapeutics and co-author of the study. “We look forward to sharing further results from this program as we continue progressing what has the potential to be a breakthrough in treating blindness in children and adults with LCA1.”

Shannon Boye, UF professor of pediatrics and Sanford Boye, associate scientist of pediatrics, and their collaborators at the University of Pennsylvania and Oregon Health and Science University published the results of the clinical trial Sept. 5 in the journal The Lancet.

LCA1 is rare. Only about 3,000 people have the condition across both Europe and the U.S. It is caused by having two defective copies of the gene GUCY2D, which is required for the light-sensitive cells in the eyes to function properly. People with the disease tend to have severely impaired vision that makes it difficult or impossible to drive, read, or navigate the world visually. 

Shannon Boye has been developing the gene therapy targeting LCA1 for more than 20 years, since she enrolled as a graduate student at UF in 2001. In collaboration with her husband Sanford Boye, Shannon Boye’s lab developed the virus-based transport system that is essential for delivering functioning copies of the GUCY2D gene into the correct cells in the eyes. The Boyes founded Atsena Therapeutics in 2019 to bring the LCA1 treatment and other gene therapies to market.

“Most pharmaceutical companies are not interested in treating these rare diseases, because they are not strong revenue generators,” Sanford Boye said. “But we think these patients deserve attention, because we have treatments that work and provide really meaningful improvements to their quality of life.”

The study enrolled 15 subjects for treatment at the University of Pennsylvania or Oregon Health and Science University. Subjects received one of three different doses of the therapy to identify the safest and most effective dose for future trials. All patients received the treatment in one eye, which involved a surgical injection in the retina.

Researchers followed the patients for a year to test their vision in the treated eye compared to the untreated eye. Subjects who received higher doses saw greater improvements in their vision. 

The researchers expect the gene therapy to last indefinitely, requiring just a single treatment per eye. So far, they have seen visual improvements last at least five years.

Broad access to the treatment will require approval by the FDA following a phase 3 clinical trial, which tests the therapy in a larger population of patients. 

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Journal

The Lancet

Method of Research

Randomized controlled/clinical trial

Subject of Research

People

Article Title

Safety and Efficacy of ATSN-101 in Patients with Leber Congenital Amaurosis caused by Biallelic Mutations in GUCY2D: A Phase 1/2, Multi-Center, Open-Label, Unilateral Dose Escalation Study

Article Publication Date

5-Sep-2024

100x improvement in sight seen after gene therapy trial

Patients with a rare inherited disease affecting their sight experienced quick vision improvements that sustained for the full year-long study

University of Pennsylvania School of Medicine

PHILADELPHIA— The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it. Some patients even experienced a 10,000-fold improvement in their vision after receiving the highest dose of the therapy, according to researchers from the Perelman School of Medicine at the University of Pennsylvania who co-led the clinical trial published in The Lancet.

“That 10,000-fold improvement is the same as a patient being able to see their surroundings on a moonlit night outdoors as opposed to requiring bright indoor lighting before treatment,” said the study’s lead author, Artur Cideciyan, PhD, a research professor of Ophthalmology and co-director of the Center for Hereditary Retinal Degenerations. “One patient reported for the first time being able to navigate at midnight outdoors only with the light of a bonfire.”

A total of 15 people participated in the Phase 1/2 trial, including three pediatric patients. Each patient had Leber congenital amaurosis as the result of mutations in the GUCY2D gene, which is essential to producing proteins critical for vision. This specific condition, which affects less than 100,000 people worldwide and is abbreviated as LCA1, causes significant amount of vision loss as early as infancy.

All subjects had severe vision loss with their best measure of vision being equal or worse than 20/80—meaning if a typically-sighted person could see an object clearly at 80 feet, these patients would have to move up to at least 20 feet to see it. Glasses provide limited benefit to these patients because they correct abnormalities in the optical focusing ability of the eye, and are unable to address medical causes of vision loss, such as genetic retinal diseases like LCA1.

The trial tested different dosage levels of the gene therapy, ATSN-101, which was adapted from the AAV5 microorganism and was surgically injected under the retina. For the first part of the study, cohorts of three adults each received one of the three different dosages: Low, mid, and high. Evaluations were held between each level of dosage to ensure that they were safe before upping the dosage for the next cohort. A second phase of the study involved only administering the high dosage levels to both an adult cohort of three and a pediatric cohort of three, again after safety reviews of the previous cohorts.

Improvements were noticed quickly, often within the first month, after the therapy was applied and lasted for at least 12 months. Observations of participating patients are also ongoing. Three of six high-dosage patients who were tested to navigate a mobility course in varying levels of light achieved the maximum-possible score. Other tests used eye charts or measured the dimmest flashes of light patients perceived in a dark environment.

Of the nine patients who received the maximum dosage, two had the 10,000-fold improvement in vision.

“Even though we previously predicted a large vision improvement potential in LCA1, we did not know how receptive patients’ photoreceptors would be to treatment after decades of blindness,” said Cideciyan. “It is very satisfying to see a successful multi-center trial that shows gene therapy can be dramatically efficacious.”

Primarily, the study sought to determine the safety of the gene therapy and its varying dosage levels. Researchers did find some patients had side effects, but the overwhelming majority were related to the surgical procedure itself. The most common side effect was conjunctival hemorrhage, the breakage of small blood vessels underneath the clear surface of the eye, which healed. Two patients had eye inflammation that was reversed with a course of steroids. No serious side effects were related to the study drug.

This work comes on the heels of another successful ophthalmological trial at Penn restoring sight in patients with a different form of LCA. Earlier in 2024, CRISPR-Cas9 gene editing was used to improve the sight of many patients with a form of LCA tied to mutations in the CEP290 gene. Co-led by one of the new paper’s co-authors, Tomas S. Aleman, MD, the Irene Heinz-Given and John LaPorte Research Professor in Ophthalmology and co-director with Cideciyan of the Center for Hereditary Retinal Degenerations, the study used similar tests and was the first time children were involved in any gene editing work.

“The treatment success in our most recent clinical trials together with our earlier experience brings hope for a viable treatment for about 20 percent of infantile blindness caused by inherited retinal degenerations,” Aleman said. “The focus now is on perfecting the treatments and treating earlier manifestations of these conditions once safety is confirmed. We hope similar approaches will lead to equally positive outcomes in other forms of congenital retinal blindness.”

Moving forward, approval of this experimental medicine for clinical use requires another trial, where participants are randomized to a treatment dose and both patients and those investigating the trial not knowing who gets what. Through that, any possible bias in results could be avoided.

This study was funded by Atsena Therapeutics, Inc. Two of the paper’s authors, Andres K. Lauer, MD, and Mark Pennesi, MD, PhD, are members of the company’s clinical and scientific advisory board.

 

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Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, excellence in patient care, and community service. The organization consists of the University of Pennsylvania Health System and Penn’s Raymond and Ruth Perelman School of Medicine, founded in 1765 as the nation’s first medical school.

The Perelman School of Medicine is consistently among the nation's top recipients of funding from the National Institutes of Health, with $550 million awarded in the 2022 fiscal year. Home to a proud history of “firsts” in medicine, Penn Medicine teams have pioneered discoveries and innovations that have shaped modern medicine, including recent breakthroughs such as CAR T cell therapy for cancer and the mRNA technology used in COVID-19 vaccines.

The University of Pennsylvania Health System’s patient care facilities stretch from the Susquehanna River in Pennsylvania to the New Jersey shore. These include the Hospital of the University of Pennsylvania, Penn Presbyterian Medical Center, Chester County Hospital, Lancaster General Health, Penn Medicine Princeton Health, and Pennsylvania Hospital—the nation’s first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is an $11.1 billion enterprise powered by more than 49,000 talented faculty and staff.

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Journal

The Lancet

Article Title

Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study

Article Publication Date

5-Sep-2024