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.

UC San Diego Awarded $8M to Expand Stem Cell Therapy Clinical Trials

UC San Diego Alpha Stem Cell Clinic awarded $8M to expand clinical trials of novel stem cell therapies. The CIRM award will advance partnerships between academic and industry experts in San Diego to expedite clinical trials for patients with difficult-to-treat diseases.

NIH Funds Miller School Researcher’s Novel Work to Develop Gene Therapy for Hearing Loss-related Usher Syndrome

The National Institute on Deafness and Other Communication Disorders (NIDCD) has awarded Xue Zhong Liu, M.D., Ph.D., Marian and Walter Hotchkiss Endowed Chair in Otolaryngology at the University of Miami Miller School of Medicine, a five-year, $3.5 million R01 research grant to develop a precision medicine approach to treat hearing loss (HL) in Usher syndrome (USH).

Children’s Hospital of Philadelphia Awards Gold Medal to Dr. Katherine High, Gene Therapy Pioneer

In an honor reserved for only the most significant achievements in advancing children’s health, Children’s Hospital of Philadelphia (CHOP) has awarded its Gold Medal to Katherine High, MD, a gene therapy pioneer and one of the lead developers of the first in vivo gene therapy approved by the Food and Drug Administration (FDA). Bestowed only 12 times in CHOP’s 167-year history, the Gold Medal highlights Dr. High’s groundbreaking discoveries at CHOP, which led to a gene therapy treatment for a rare form of inherited blindness and advanced gene therapy for hemophilia to late-stage testing.

Cardiologists, Cardiothoracic Surgeons Available for Expert Commentary Ahead of Upcoming Conferences

Cardiologists and cardiothoracic surgeons from the Smidt Heart Institute at Cedars-Sinai are available to discuss the latest advances in research, clinical care, transcatheter procedures and cardiothoracic surgery throughout the International Society for Heart and Lung Transplantation (ISHLT) Scientific Sessions 2022 and Heart Rhythm 2022.

UCI receives 5-year, $5 million CIRM award for training of diverse researchers

The University of California, Irvine has received a five-year, $5 million award from the California Institute for Regenerative Medicine to support a comprehensive doctoral, postdoctoral and clinical researcher training program to prepare the current and next generation of leaders in stem cell biology, gene therapy and regenerative medicine.