Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Gene editing within a living person for the first time

Genetic editing has been performed in the laboratory until now. Living cells within Petri-dishes have been put under the microscope while gene editing tools such as CRISPR were used to cut, nick and replace parts of the DNA or genome that is defective with bits of DNA that is correct in its sequence. Now doctors in California have attempted to edit cells within a living person.

19 Nov 2017

Study finds genetic treatment for inherited retinal disorder

A new study presented at the 121st Annual Meeting of the American Academy of Ophthalmology (AAO) 2017, for the first time found a novel gene therapy that can improve the eyesight of patients with inherited retinal disease.

13 Nov 2017

New gene therapy for blindness may soon be reality

Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting of the American Academy of Ophthalmology.

10 Nov 2017

Complete new skin from transgenic stem cells for boy with rare genetic disease

Hassan, a then seven-year-old boy from Germany, in 2015, presented to the doctors with a rare skin disease called junctional epidermolysis bullosa or JEB. This condition leaves the skin thin and papery and easily breakable. It has been compared to “butterfly wings” because of their fragile nature.

8 Nov 2017

New gene therapy trial for X-linked myotubular myopathy offers hope to children

Ann & Robert H. Lurie Children's Hospital of Chicago is one of the few centers participating in ASPIRO, an international Phase 1/2 clinical trial of a gene therapy product called AT132 for X-linked myotubular myopathy – a rare disease characterized by severe muscle weakness, breathing difficulty and early death.

8 Nov 2017

Study solves critical piece of deafness puzzle by identifying long non-coding RNAs

A new Tel Aviv University study solves a critical piece of the puzzle of human deafness by identifying the first group of long non-coding RNAs (lncRNAs) in the auditory system.

2 Nov 2017

Conference to focus on preventing and reversing consequences of sickle cell disease

Leading experts in the field of sickle cell disease (SCD) research will convene in Washington, D.C., for the Physiological and Pathophysiological Consequences of Sickle Cell Disease conference (November 6–8). The conference, organized by the American Physiological Society, will explore SCD- the world's most prevalent single-gene mutation disease-; and new research on preventing and reversing its deadly consequences.

1 Nov 2017

Gene therapy prevents age-related learning and memory problems in mice

Researchers from the Institute of Neurosciences at the Universitat Autònoma de Barcelona and the Vall d'Hebron Research Institute are the first to demonstrate that regulation of the brain's Klotho gene using gene therapy protects against age-related learning and memory problems in mice.

31 Oct 2017

Clinical trial shows promising results for treatment of patients with recurrent high-grade glioma

New data from a Phase I clinical trial led by Clark Chen, M.D., Ph.D., Lyle French Chair in Neurosurgery and Head of the University of Minnesota Medical School Department of Neurosurgery shows more than a quarter of patients with recurrent high-grade glioma, a form of brain cancer, were alive more than three years after treatment.

28 Oct 2017

Research advance may lead to preservation of hearing in people with Usher syndrome type III

A research advance co-led by Case Western Reserve University School of Medicine's Kumar Alagramam, PhD, may stop the progression of hearing loss and lead to significant preservation of hearing in people with Usher syndrome type III, a form of hereditary hearing loss linked to defects in the sensory "hair" cells in the inner ear. USH3 is caused by a mutation in the clarin-1 gene.

26 Oct 2017

VICC selected to administer FDA-approved CAR T therapy for treatment of lymphoma patients

Vanderbilt-Ingram Cancer Center has been selected as one of the few authorized treatment centers in the United States approved to administer the first FDA- approved chimeric antigen receptor T cell (CAR T) therapy for treatment of adult patients with a specific type of lymphoma.

25 Oct 2017

Scientists receive critical funding to study treatments for three deadly cancers

Immunotherapy for leukemia patients has been nothing short of a miracle. Now scientists hope to use that science and other forms of gene therapy to tackle three of the deadliest forms of cancer: glioblastoma (brain cancer), sarcoma (bone cancer) and ovarian cancer.

17 Oct 2017

Cascade of costs could push new gene therapy above $1 million per patient

Outrage over the high cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the country's first gene therapy, Novartis' Kymriah, a leukemia treatment approved in August.

17 Oct 2017

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

A panel of advisors to the United States Food and Drug Administration (FDA) has voted unanimously in favor of an experimental gene therapy to treat patients with a rare kind of hereditary blindness.

16 Oct 2017

MIT researchers develop new technique to screen for genes that protect against diseases

Using a modified version of the CRISPR genome-editing system, MIT researchers have developed a new way to screen for genes that protect against specific diseases.

12 Oct 2017

New microcapsules represent promising vectors for application of genome-editing tools

Researchers from Tomsk Polytechnic University jointly with their colleagues from St. Petersburg, Hamburg and London have conducted a study in the course of which it was found out that polymer and hybrid silica-coated microcapsules are more efficient in genome-editing when applying CRISPR-Cas9 system.

6 Oct 2017

CGT Catapult’s iPSC line licensed to Universal Cells for creating universal donor cell line

The Cell and Gene Therapy Catapult today announced a non-exclusive licensing agreement to provide its clinical-grade induced Pluripotent Stem Cell line to Seattle based Universal Cells.

6 Oct 2017

Moms of children with rare genetic illness push for wider newborn screening

Kerri De Nies received the news this spring from her son's pediatrician: Her chubby-cheeked toddler had a rare brain disorder.

5 Oct 2017

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

In a recent clinical trial, a gene therapy to treat cerebral adrenoleukodystrophy (CALD) -- a neurodegenerative disease that typically claims young boys' lives within 10 years of diagnosis -- effectively stabilized the disease's progression in 88 percent of patients, researchers from the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Massachusetts General Hospital report today.

4 Oct 2017

New gene therapy to treat cancer in children, young adults offers hopes and challenges

The first gene therapy for cancer, approved by the Food Drug Administration in August, will transform the treatment of a particular kind of cancer in children and young adults. It's transformative because it uses a patient's own immune cells to attack the cancer cells.

3 Oct 2017