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UrduAn 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).
Tag: Gene Therapy
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.
Scientists Receive $4.8M to Pursue Gene Therapy for ‘Incurable’ Disease
The California Institute for Regenerative Medicine has awarded UC San Diego researchers $4.8 million to advance a gene therapy to treat Friedreich’s ataxia, a rare but devastating neuromuscular disorder.
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.
A link between lethal childhood disease and age-related muscle decline
Adopting some of the strategies behind successfully treating the childhood disease spinal muscular atrophy may enable development of therapies to curb the muscle decline that accompanies aging, new research suggests.
Silicon nanochip could treat traumatic muscle loss
Technology that can change skin tissue into blood vessels and nerve cells also shows promise as a treatment for traumatic muscle loss.
Proof-of-Concept Study Advances Potential New Way to Deliver Gene Therapy
Johns Hopkins Medicine researchers say they have successfully used a cell’s natural process for making proteins to “slide” genetic instructions into a cell and produce critical proteins missing from those cells.
United BioChannels Acquires ORFLO Technologies to Expand Market Share and Enhance Customer Engagement
United BioChannels (UBC), a commercial strategy consultancy, announced its acquisition of ORFLO Technologies, LLC, from Gemini Bioproducts.
Gene Therapy Rapidly Improves Night Vision in Adults with Congenital Blindness
Patients’ low-light sensitivity improved by factors of thousands in a clinical trial
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).
Gene Therapy Approach Shows Promise in Treating ALS
Researchers report that a gene therapy approach, developed at UC San Diego measurably delayed disease onset in humanized mouse and rat models of familial ALS, an inherited form of the disease that runs in families.
“Soft” CRISPR May Offer a New Fix for Genetic Defects
Scientists have developed a CRISPR-based technology that could offer a safer approach to correcting genetic defects. The new “soft” CRISPR system makes use of natural DNA repair machinery, providing a foundation for novel gene therapy strategies with the potential to cure genetic diseases.
Mouse study shows gene therapy may correct creatine deficiency disorder
A new study in mice finds that a gene therapy developed by a UCLA researcher appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements and recurrent seizures.
Scientists Gain Ground on Rare Congenital Neurological Disorder
Two recent discoveries co-led by scientists at Cedars-Sinai may help lead to new ways to treat patients with Allan-Herndon-Dudley syndrome (AHDS), a brain development disorder that causes severe intellectual disability and problems with movement.
Change of temperature causes whole body reprogramming
Human beings, like most organisms, are constantly exposed to alternating colder or warmer temperatures.
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.
Gene Therapy Could Treat Pitt-Hopkins Syndrome, Proof-of-Concept Study Suggests
University of North Carolina School of Medicine scientists, who report their results in the journal eLife, devised an experimental, gene-therapy-like technique to restore the normal activity of the gene deficient in people with Pitt-Hopkins syndrome.
Gene Therapy Reverses Effects of Autism-Linked Mutation in Brain Organoids
UC San Diego scientists use lab-grown human brain tissue to identify neural abnormalities in Pitt-Hopkins Syndrome and show gene therapy tools can rescue neural structure and function.
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.
Gene Therapy Shows Early Promise as Angelman Syndrome Treatment
Scientists publish encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities.