Advancing Care of a Devastating Disease Through Gene Therapy

A new international trial is investigating the efficacy and safety of gene therapy in patients with Hurler syndrome and will compare results of gene therapy against BMT which is the current standard of care. UCSF Benioff Children’s Hospitals are among three sites in the U.S. and seven in the world now enrolling patients for the clinical trial.

$14M NIH grant funds gene-editing research for rare metabolic diseases at Penn and CHOP

A $14M grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children’s Hospital of Philadelphia (CHOP). The research will focus specifically on developing therapies for urea cycle disorders, which impact roughly 1 in every 35,000 children.

Improving Access to Cell and Gene Therapy in Pediatrics

Nearly 30 years ago, Children’s Hospital Los Angeles supported one of the first clinical trials for a pioneering stem cell gene therapy for children with a life-threatening form of severe combined immunodeficiency (SCID). Called ADA-SCID—a type of “bubble boy disease” caused by mutations in the adenosine deaminase (ADA) gene—this rare disorder leaves babies with virtually no immune system.

Single-Dose Gene Therapy is Potentially Life-Changing for Adults with Hemophilia B

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of Pennsylvania Perelman School of Medicine and a multicenter group of investigators.

MinJun Kim awarded NSF grant for nanosensor technology that improves gene therapy

Nanotechnology expert MinJun Kim, the Robert C. Womack Endowed Chair Professor at SMU Lyle School of Engineering, and his research team have been awarded a $300,000 grant from the National Science Foundation to design a nanosensor that can improve the accuracy of gene therapy, enabling more effective clinical trials with fewer side effects.

100x Improvement in Sight Seen After Gene Therapy Trial

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.

Pharmacy professor receives $1.9M award to tackle gene therapy challenges

Assistant Professor of Pharmaceutical Sciences Kuo-Ching (KC) Mei from Binghamton University, State University of New York and his team have won the $1.9M Maximizing Investigators’ Research Award (MIRA) for Early Stage Investigators (ESI) from the National Institute of General Medical Sciences to tackle gene therapy challenges.

Advancing regenerative medicine therapies

University and community guests recently gathered to celebrate the 7,700 square-foot Good Manufacturing Practice facility’s grand opening in Hewitt Hall’s basement on the UC Irvine campus. Although the highly sterile environment remains off-limits for tours, the gala highlighted its importance to the campus, the UC system and the local community.

The American Macular Degeneration Foundation Showcases Breakthrough AMD Research at ARVO 2024

The American Macular Degeneration Foundation supports a diverse portfolio of research investigations to advance the development of treatments, tools and usable information that improve the lives of those affected by AMD.

Rutgers-Led Statewide Translational Research Institute Is Awarded $39.7 Million National Institutes of Health Grant

The National Institutes of Health (NIH) has awarded the Rutgers Institute for Translational Medicine and Science $39,673,786 over seven years to build and improve upon infrastructure that promotes clinical and translational science through the New Jersey Alliance for Clinical and Translational Science (NJ ACTS).

A Revolution In Gene Therapy Is Unfolding, Bringing Hope for Vision Loss

A new approach to gene therapy resets cell homeostasis. Clinical trials to treat the retinal disorders Retinitis Pigmentosa, Stargardt’s, and Geographic Atrophy (late dry macular degeneration) are underway and showing promise.

Gene therapy offers hope for giant axonal neuropathy patients

A gene therapy developed by researchers at UT Southwestern Medical Center for a rare disease called giant axonal neuropathy (GAN) was well tolerated in pediatric patients and showed clear benefits, a new study reports. Findings from the phase one clinical trial, published in the New England Journal of Medicine, could offer hope for patients with this rare condition and a host of other neurological diseases.

Penn scientists create novel technique to form human artificial chromosomes

Human artificial chromosomes (HACs) capable of working within human cells could power advanced gene therapies, including those addressing some cancers, along with many laboratory applications, though serious technical obstacles have hindered their development. Now a team led by researchers at the Perelman School of Medicine at the University of Pennsylvania has made a significant breakthrough in this field that effectively bypasses a common stumbling block.

Companion Penn Medicine Studies on AAV-based Gene Therapies in Non-Human Primates Suggest Integration into Human DNA is Unlikely to Drive Cancer Mutations While Offering the Potential for Durable Expression of the Transgene

Gene therapy adeno-associated viruses (AAVs)—viruses that can be engineered to deliver DNA to target cells—are unlikely to cause cancer-triggering insertions in humans or monkeys and may contribute to long-term efficacy, according to new research from the University of Pennsylvania’s Gene Therapy Program (GTP).

Wexner Medical Center among first in nation to administer new gene therapy for ALS

The Ohio State University Wexner Medical Center is one of the first nationwide to administer a targeted gene therapy for patients with a specific form of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

UC Davis Eye Center tests experimental gene therapy for wet age-related macular degeneration (AMD)

Ophthalmologists at UC Davis Health used an experimental gene therapy last month to treat a patient with wet age-related macular degeneration, or wet AMD. Wet AMD is a leading cause of vision loss among older adults. Glenn Yiu, a professor of ophthalmology at UC Davis Health, is the principal investigator for the new clinical trial.

CHOP and Penn Medicine Researchers Develop “In Vivo” RNA-based Gene Editing Model for Blood Disorders

In a step forward in the development of genetic medicines, researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body. If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant. The findings were published today in the journal Science.

Novel Gene Therapy Proving Safe and Successful in Sickle Cell Patients Treated at Cleveland Clinic Children’s

Researchers presenting preliminary data from a clinical trial aimed at discovering a cure for sickle cell disease reveal positive results among its first patients. Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies.

IIT Kanpur pioneers ground-breaking gene therapy technology for Hereditary Eye Diseases

IIT Kanpur has licensed a pioneering technology to Reliance Life Sciences Pvt. Ltd. that has the potential to revolutionize the field of gene therapy, especially for many genetic eye diseases, making it the first time that a gene therapy related technology has been developed and transferred from an academic institution to a company in India.

Penn Medicine at the American Society of Gene and Cell Therapy 26th Annual Meeting

Researchers from the Gene Therapy Program (GTP) at the Perelman School of Medicine at the University of Pennsylvania will present nine abstracts highlighting their translational science and discovery research on gene therapy, gene editing, and adeno-associated virus (AAV) vector technology at the American Society of Cell and Gene Therapy (ASGCT) 26th Annual Meeting on May 16–20, 2023 in Los Angeles, California.

Improving gene therapy with tiny bubbles

Beyond vaccines, mRNA offers immense potential to fight disease, but targeting the genetic material to specific diseased cells is challenging—requiring a new method. To meet this need, researchers at the Case Western Reserve University School of Medicine, with a Global Research Fellowship award from Moderna Inc., are developing a process that essentially uses bubbles to overcome the problem.

新的基因编辑技术成功地逆转小鼠的视力损失

视网膜色素变性症是人类失明的主要原因之一。中国的研究人员成功地恢复了患有视网膜色素变性症的小鼠的视力。该研究将于[三月十七日]发表在《实验医学杂志》上。该研究使用一种新型的、高度通用的CRISPR基因组编辑技术,有潜力纠正各种导致疾病的遗传突变。

New gene-editing technique reverses vision loss in mice

Researchers in China have successfully restored the vision of mice with retinitis pigmentosa, one of the major causes of blindness in humans. The study, to be published March 17 in the Journal of Experimental Medicine, uses a new, highly versatile form of CRISPR-based genome editing with the potential to correct a wide variety of disease-causing genetic mutations.

ARVO Foundation Announces 2022 Point of View Award Winner

Rockville, Md.—The Association for Research in Vision and Ophthalmology (ARVO) congratulates Tasneem Khatib DM, FRCOphth—recipient of the 2022 Point of View Award.

Established by the Point of View Foundation (Fundació Punt de Vista), the award provides a $20,000 cash prize in recognition of an outstanding scholarly article related to efforts to restore vision through regenerative ophthalmology, biotechnology, whole eye transplantation or other approaches.

ARVO Foundation Names 2022 Recipient of Kreissig Award for Excellence in Retinal Surgery

Rockville, Md.—The Association for Research in Vision and Ophthalmology (ARVO) Foundation congratulates Demetrios G. Vavvas, MD, PhD, the 2022 recipient of the Kreissig Award for Excellence in Retinal Surgery. Established by Ingrid Kreissig, MD, through the ARVO Foundation, the $30,000 award recognizes excellence in the understanding of the mechanisms of retinal disease requiring surgical management, innovative approaches to management, and/or outcomes of surgical treatment.