Sentences with phrase «gene therapy trials for»

The vector is a self - inactivating lentivirus — a member of a family of viruses that can insert genes into mammalian cells and drive expression of those genes — that has been engineered to avoid triggering the development of leukemia, a complication seen in previous gene therapy trials for immunodeficiency syndromes, including WAS.

WOMEN with cystic fibrosis are to join British gene therapy trials for the first time.

AAV1 is considered safe as a viral vector and is already in use in human gene therapy trials for blindness, heart disease, muscular dystrophy and other conditions.

IN TWO months» time, a group of profoundly deaf people could be able to hear again, thanks to the world's first gene therapy trial for deafness.

(In the early 2000s, five children who participated in a retrovirus - based gene therapy trial for severe combined immunodeficiency developed leukemia.)

Researchers say they see promising results from the first gene therapy trial for Alzheimer's disease.

After preclinical studies, a gene therapy trial for SCID - X1 was initiated, based on the use of complementary DNA containing a defective γc Moloney retrovirus — derived vector and ex vivo infection of CD34 + cells.

The «transformational» results have particular significance as the first successful gene therapy trial for the haemophilia A.

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott - Aldrich syndrome (WAS), a life - threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology.

Not exact matches

While it takes time for gene therapies to be tested, trialed, and possibly approved by the FDA, genetics startups like Spark, AveXis, Caribou Biosciences, Editas, and Audentes have been on the road a while.

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.

Human trials are even costlier, so for now, Sweeney says, IGF - 1 and myostatin gene therapies remain on the distant horizon.

The trial he conducted tested the safety of a therapy for ornithine transcarbamylase (OTC) deficiency, a rare disorder in which the liver lacks a functional copy of the OTC gene.

The Food and Drug Administration immediately terminated all gene therapy trials there, and the incident prompted federal regulators to establish new rules for human gene therapy research.

«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.

Now Aubourg and his team have showed in a preliminary trial that gene therapy stopped ALD in two boys for whom they could not find matching bone marrow donors.

Two of 10 children treated with gene therapy for SCID in a French trial develop leukemia, researchers announced in 2002, and it is discovered that the virus had inserted genes in several unexpected places around the genome, leading the cells to become cancerous.

Regulators in the US could soon be asked to approve a human trial of gene therapy for cystic fibrosis that uses a hybrid of the HIV and Ebola viruses.

«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..

Some on the panel suggested they were particularly sensitive about such concerns given that it was at UPenn in 1999 that a young man, Jessie Gelsinger, died in a gene therapy trial, setting the field back for years.

Gene therapy death The Food and Drug Administration stopped a gene therapy trial this week after a patient undergoing the experimental treatment for a severe form of arthritis had a fatal reactGene therapy death The Food and Drug Administration stopped a gene therapy trial this week after a patient undergoing the experimental treatment for a severe form of arthritis had a fatal reactgene therapy trial this week after a patient undergoing the experimental treatment for a severe form of arthritis had a fatal reaction.

«First trial of gene therapy for cystic fibrosis to show beneficial effect on lung function.»

A therapy that replaces the faulty gene responsible for cystic fibrosis in patients» lungs has produced encouraging results in a major UK trial.

(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.

Jonathan Appleby, GSK project leader for the ADA - SCID trial and head of cell and gene therapy at the rare diseases unit, says there are many differences between working on this kind of trial and working with one for a common disease.

Although several experimental trials of gene therapy are under way, some of them for more than a decade, uniQure is the first in the West to win regulatory approval for a commercial treatment.

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.

The LCA2 trials are a rare success for the field of gene therapy, which has also cured children with the immune disorder known as bubble boy disease.

As for worries about harmful effects, researchers say AAV8 isn't likely to disrupt healthy genes, a side effect that stalled a promising gene - therapy trial for an immune - deficiency disorder in 2002.

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

His story inspired a book, The Forever Fix, which heralded the LCA trials as part of the wave of successes for gene therapy, which was recovering from several setbacks — including the death of a patient and cancer in others — in the late 1990s and early 2000s.

Although the study's approach wasn't a sure bet, the researchers hoped to go further than any other gene therapy trial yet for this relatively common inherited disease that fills people's lungs with sticky mucus that promotes deadly infections.

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

«It is a tough calculus, but gene therapy for CF lung disease is still a realistic possibility and studies should be encouraged — especially well - done ones like the U.K. trial.

A «cure» for haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led by the NHS in London.

These are skills that we implement for other international trials of gene therapy for rare genetic diseases of the immune system, blood, muscle, vision or liver... We will continue the current study with the objective of providing treatment for patients.»

However, questions about the design and ethics of the trial have cast a negative light on the field, particularly gene therapy for non-life-threatening diseases.

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.

And the participants will be monitored for 15 years for health impacts, as is required by the FDA for any gene - therapy trials.

Other blood disorders that have shown significant benefit from targeted gene therapy in small trials include hemophilia (specifically, factor IX deficiency), severe beta - thalassemia (deficiency for the adult beta - globin gene) and leukemia, where the patient's immune cells were treated to enable them to recognize cancer cells and destroy them.