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.
Tag: Gene Therapy
New Gene Therapy for Retinitis Pigmentosa Shows Promise
New research suggests that a new kind of gene therapy can improve vision in people who have lost nearly all sight to retinitis pigmentosa.
$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.
What Is Gene Therapy?
Every cell in your body holds a unique genetic code within your DNA, inherited from your parents. The segments of your DNA called genes determine nearly all your body’s characteristics and functions.
New gene therapy approach shows promise for Duchenne muscular dystrophy
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).
New gene therapy for muscular dystrophy offers hope
The UW Medicine-led research focuses on delivering a series of protein packets inside a shuttle vector to replace the defective DMD gene within the muscles. The added genetic code will then start producing dystrophin, the protein lacking in patients with muscular dystrophy.
UNC Researchers Identify Potential Treatment for Angelman Syndrome
Researchers at the UNC School of Medicine have identified a small molecule that could lead to a safe and effective treatment for the neurodevelopmental condition known as Angelman syndrome.
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.
CellFE Announces Addition of Life Science Tools Veteran Mike Rice to Board of Directors
CellFE Inc., a cell engineering platform company with a novel microfluidic technology for non-viral cell therapy manufacturing, announced today that life science tools executive Mike Rice has been appointed to its Board of Directors.
Gene therapy relieves back pain, repairs damaged disc in mice
Disc-related back pain may one day meet its therapeutic match: gene therapy delivered by naturally derived nanocarriers that, a new study shows, repairs damaged discs in the spine and lowers pain symptoms in mice.
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.
Researchers Publish Final Results of Key Clinical Trial for Gene Therapy for Sickle Cell Disease
An international consortium published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
Lurie Children’s Hospital First in Illinois to Treat Duchenne Muscular Dystrophy with FDA-Approved Gene Therapy
On March 27, 2024, Ann & Robert H. Lurie Children’s Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy – a rare, genetic disease characterized by progressive muscle damage and weakness.
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.
Researchers are using RNA in a new approach to fight HIV
Society learned about the value of mRNA during the COVID-19 pandemic when we saw scientists and medical professionals harness its power to deliver a vaccine for the virus within a year.
Junk DNA in birds may hold key to safe, efficient gene therapy
The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease.
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).
New methods for effective transport of large genes in gene therapy
Gene therapy currently represents the most promising approach for the treatment of hereditary diseases. Yet despite significant breakthroughs in recent years, there are still a number of hurdles that hinder the wider application of gene therapies.
Pursuing Three Gene Therapies for Rare Inherited Disease
Researchers hope at least one will treat progressive blindness caused by Usher syndrome type 1F
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.
Gene therapy for brain tumor shows promising early results in humans
A new study from the University of Michigan Department of Neurosurgery and Rogel Cancer Center shows promising early results that a therapy combining cell-killing and immune-stimulating drugs are safe and effective in extending survival for patients with gliomas, a highly aggressive form of brain cancer.
New study shows promising evidence for sickle cell gene therapy
UChicago Medicine Comer Children’s Hospital was one of three sites to enroll patients in a clinical trial to test a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.
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.
Gene therapy study identifies potential new treatment for liver cancer
Gene therapy with microRNA-22 produced better survival outcomes than the current FDA-approved drug for liver cancer and without noticeable toxicity in new mouse study.
Research sheds new light on gene therapy for blood disorders
A study shows new steps toward more patients getting gene therapy
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.
Base editing shows potential superiority for curing sickle cell disease
Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease and beta thalassemia. Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard found base editing increased fetal hemoglobin production in a new treatment.
Diagnosis of rare, genetic muscle disease improved by new approach
It’s not easy to distinguish between the dozens of subtypes of limb girdle muscular dystrophy — a rare, genetic muscle disease characterized by weakness in the hips and shoulders that causes difficulty walking and lifting the arms.
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.
Analysis: Gene therapy for sickle cell disease will be cost-effective if health disparities considered
New multimillion-dollar treatments for sickle cell disease (SCD) will be cost-effective in the U.S. if racial health outcome and health access disparities are taken into consideration, according to a new cost analysis led by Yale School of Medicine’s George Goshua,…
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.
Toward a Therapy for a Rare Genetic Disease
Researchers design “mini gene” therapy for severe syndrome that causes blindness and deafness
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.
Step Forward in Gene Therapy to Treat Cause of Sudden Cardiac Arrest in Athletes
University of Utah Health scientists have corrected abnormal heart rhythms in mice, suggesting a new strategy for treating arrhythmogenic cardiomyopathy, the leading cause of cardiac arrest in young athletes.
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.
U-CARS 2023: Healing Diseased Hearts, from Bench to Bedside
Now in its 11th year, participants in Utah Cardiac Recovery Symposium (U-CARS) will exchange ideas and evaluate paradigms on a now-thriving field of science and medicine that was once thought to be impossible: making diseased hearts healthy again.
University of Utah and TikkunLev Therapeutics announce new partnership to accelerate heart-failure gene therapy
The new partnership aims to accelerate an innovative heart-failure gene therapy. The agreement is an exclusive world-wide license and includes a sponsored research program to support future FDA filings.
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.
Liver cancer study encourages caution with certain gene therapies
A newly discovered link between protein misfolding and liver cancer could help improve gene therapy for hemophilia.