Sentences with phrase «gene therapy clinical trials for»

He notes that AAV is already being used in gene therapy clinical trials for Parkinson's disease, hemophilia, and vision disorders.

11/10/2008 UC San Diego Medical Center Offers Gene Therapy Clinical Trial for Advanced Heart Failure UC San Diego Medical Center is currently enrolling patients in a Phase 2 clinical trial of an investigational drug for the treatment of advanced heart failure.

Not exact matches

Katherine High, Spark's president and chief scientific officer, expressed her enthusiasm for the early clinical data related to SPK - 8011: «The encouraging start of our SPK - 8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof - of - concept in a second liver - mediated disease — a significant achievement in the gene therapy field — and positions us well to potentially transform the current treatment approach for this life - altering disease with a one - time intervention.»

He and his lab, for example, along with the company Neurologix based in Fort Lee, N.J., are conducting a clinical trial to test a similar gene - therapy treatment for Parkinson's disease.

These allusions to the past aren't surprising considering how drastically the clinical trial changed gene therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for Human Gene Therapy, where the test took plgene therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for Human Gene Therapy, where the test tooktherapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for Human Gene Therapy, where the test took plGene Therapy, where the test tookTherapy, where the test took place.

«My team at Nationwide Children's has worked with commitment and dedication to develop a therapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide Chiltherapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide ChilTherapy at Nationwide Children's.

Although gene therapy research has made great strides in recent years, it has yet to be widely deployed, and no CRISPR - edited genes have yet been tested for safety or efficacy in human clinical trials.

AAV is in use in many late - stage clinical trials in the United States, and has already been approved for use in one gene therapy drug in the European Union.

This study represents a significant step towards the development of clinical trials in gene therapy for the curative treatment of hereditary deafness and balance loss in humans.

AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXStherapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXSTherapy Designation for the treatment based on preliminary clinical results from the trial of AVXS - 101.

«Fibroblast growth factor receptor inhibitors are new therapies being developed in clinical trials for patients whose cancer cells have genetic alterations in this family of genes,» says Roychowdhury, a member of the OSUCCC — James Translational Therapeutics Program.

«Our gene therapy protocol is not yet ready for clinical trials — we need to tweak it a bit more — but in the not - too - distant future we think it could be developed for therapeutic use in humans,» says Jeffrey Holt, PhD, a scientist in the Department of Otolaryngology and F.M. Kirby Neurobiology Center at Boston Children's and an associate professor of Otolaryngology at Harvard Medical School.

It showed effectiveness against liver cancer in a phase II clinical trial and will move into a phase III trial later this year, David Kirn, an oncologist and the company's president and chief executive officer, said at a recent meeting of the American Society for Gene & Cell Therapy in Washington, D.C..

(Adeno - associated virus or AAV has been approved in Europe as a vector for gene therapy, and is being explored in the United States for clinical trials.)

The team envisions that in the future, deaf people might benefit from gene therapy similar to the approaches currently being tested in clinical trials for other diseases.

As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist - oncologist from Dana - Farber / Boston Children's Cancer and Blood Disorders Center argues in a commentary published by the journal Science.

Boston Children's Hospital has offered non-exclusive licenses to for - profit entities on a patent developed by Orkin's laboratory regarding BCL11A, a genetic switch regulating hemoglobin production that is expected to form the basis of clinical trials for gene therapy and gene editing for sickle cell disease and thalassemia.

Woo and Ledley predict that clinical trials using gene therapy for metabolic disorders such as PKU could start within eight years.

Nonetheless, the result has renewed concern about the oversight of clinical trials for gene therapy.

For very rare diseases such as WAS, multicenter clinical trials are the only effective way of proving the safety and efficacy of gene therapy and having it rapidly approuved and made available to all patients.

«RNAi therapies are a unique approach to cancer treatment as they have the potential to «turn off» the genes» coding for proteins involved in cancer cell division,» said Ramesh K. Ramanathan, M.D., medical director of the Virginia G. Piper Cancer Center Clinical Trials Program at Scottsdale Healthcare and deputy director of the Clinical Translational Research Division of the Translational Genomics Research Institute (TGen) in Phoenix, Ariz. «Using a lipid nanoparticle, the RNAi drug can be delivered to a cancer cell to block the expression of specific proteins involved in tumor growth.»

Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler - Najjar SyndGene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler - Najjar Syndgene therapy treatment for a rare liver disease, Crigler - Najjar Syndrome

UC San Diego Health joins national clinical trial testing a gene therapy treatment for individuals with hemophilia B.

Colleen Dansereau, RN, MSN, CPN Program Manager, Gene Therapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGene Therapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseaseTherapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseaseTherapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseasetherapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (Cgene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous diseasetherapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGD).

Although this field is still relatively young, evidence for clinical efficacy has been observed and continued progress seems assured, as clinical trials continue to yield insights into how gene therapy can be applied and improvements are made in gene therapy tools.

Weill Cornell is the birthplace of many medical advances — including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo - biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson's disease, and most recently, the world's first successful use of deep brain stimulation to treat a minimally conscious brain - injured patient.

Spark Therapeutics announced yesterday that they will be taking a gene therapy treatment for LHON (Leber Hereditary Optic Neuropathy) into clinical trials.

Spark Therapeutics» gene therapy for patients with haemophilia has seen some early success in clinical trials, after being tested in 10 male patients

Spark Therapeutics» gene therapy for patients with haemophilia has seen some early success in clinical trials, according to a report published yesterday.

AFM supports each year more than 400 research programs, among them more than 30 clinical trials in gene therapy or cellular therapy concerning for example muscular, eyes, skin or blood diseases.

CAR T - cell Therapy: Scott McIntyre's Story After many treatments for his B - cell lymphoma failed, Scott McIntyre became the first UChicago Medicine patient to undergo CAR T - cell gene therapy in a clinicalTherapy: Scott McIntyre's Story After many treatments for his B - cell lymphoma failed, Scott McIntyre became the first UChicago Medicine patient to undergo CAR T - cell gene therapy in a clinicaltherapy in a clinical trial.

Yesterday a team of University of Pennsylvania researchers — led by Dr Pablo Tebas, Professor Carl June, and Dr Bruce Levine — announced the successful conclusion of a clinical trial to evaluate the safety of a new gene therapy technique for treating HIV.

Paris, France, April 3, 2018, 5.35 pm CET — GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA - PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced topline results from the REVERSE Phase III clinical trial evaluating the safety and efficacy of a single intravitreal injection of GS010 (rAAV2 / 2 - ND 4) in 37 subjects whose visual loss due to 11778 - ND4 Leber Hereditary Optic Neuropathy (LHON) commenced between 6 and 12 months prior to study treatment.

That clearance would be a high - water mark for gene therapy research, which suffered a major setback in 1999 with the death of a patient in a clinical trial for a liver disorder.

Reid and his colleagues undertook this study following the death of Jesse Gelsinger, a participant in a gene therapy clinical trial at University of Pennsylvania for ornithine transcarbanoylase (OTC) deficiency, a metabolic liver disorder.

Indeed, Leber Congenital Amaurosis (LCA) was one of the first recessive disorders to undergo clinical trials for gene replacement therapy.

Lysogene's MPS IIIA gene therapy clinical trial strategy includes a partnership with the MPS patient networks to ensure families are well informed about our development program and are able to make informed choices concerning the best treatment strategy for their children.

«Our work in NPC1 mice may help lead to human clinical trials and eventually FDA approval for gene therapy as a treatment for NPC1 disease,» said Charles P. Venditti, M.D., Ph.D., senior investigator in the NHGRI Medical Genomics and Metabolic Genetics Branch.

Auburn University College of Veterinary Medicine, UMass Medical School to test gene therapy treatment for Tay - Sachs and Sandhoff diseases Researchers at Auburn University College of Veterinary Medicine and UMass Medical School are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.

New treatments are being tested to tackle the crippling difficulties of vertigo, including a clinical trial of prosthetic ear implants and ear gene therapy, with initial work revealing novel aspects of brain anatomy linked to balance which could be used as targets for future treatments.