Sentences with phrase «develop gene and cell therapies»

The CRISPR - Cas technology can be used to screen for druggable targets and to develop gene and cell therapies for unmet medical needs.

Not exact matches

US - based pharmaceutical company Gilead Sciences entered the chimeric antigen receptor (CAR) T - cell therapy business through its acquisition of Kite Pharma, and Australian biopharma company CSL Behring acquired US - based Calimmune, a company that develops clinical - stage gene therapy solutions.

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.

For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration, pointed to an array of promising NIH activities, including the development of new technologies to provide insights into human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct mutations and clear the way to develop and test a «curative therapy» for the first molecular disease: sickle cell disease.

Researchers at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital and Houston Methodist have developed an alternative treatment in which virus - specific cells protect patients against severe, drug - resistant viral infections.

«Researchers ID cancer gene - drug combinations ripe for precision medicine: Yeast, human cells and bioinformatics help develop one - two punch approach to personalized cancer therapy.»

In a step toward accelerating the production of new gene therapies, scientists report in ACS Nano that they have developed remote - controlled, needle - like nanospears capable of piercing membrane walls and delivering DNA into selected cells.

Dr. Levine directs the Clinical Cell and Vaccine Production Facility (CVPF), which develops, manufactures, and tests novel cell and gene therapies in clinical trials at Penn and collaborating institutiCell and Vaccine Production Facility (CVPF), which develops, manufactures, and tests novel cell and gene therapies in clinical trials at Penn and collaborating instituticell and gene therapies in clinical trials at Penn and collaborating institutions.

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.

In recent years, scientists have grown new retinal cells from stem cells and shown progress in developing an effective gene therapy.

Amongst others, Dr. Sonntag's work has contributed to developing a cell therapy for Parkinson's disease (PD), and to understanding a role of dysregulated gene and miRNA expression networks in PD and schizophrenia, or dopaminergic and adrenergic receptors in hedonic and impulsive behavior.

Intellia is developing human gene and cell therapies for both ex vivo and in vivo applications using CRISPR - Cas9 gene editing technology.

A physician with a longstanding interest in gene therapy, Dr. High was formerly a Professor at the Perelman School of Medicine of the University of Pennsylvania, an Investigator of the Howard Hughes Medical Institute, and the Founding Director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, a Center focused on developing novel cell and gene - based therapies for genetic disease.

The company is developing CAR T - cell immunotherapies for multiple myeloma, prostate and other cancer types, as well as gene therapies for orphan diseases.

While the vast majority of research in this field is focused on developing stem cell, gene and tissue engineering therapies, these approaches have not yet delivered on their promise despite nearly two decades of research.

It's about co-funding industrial facilities for developing and producing gene and cell therapies, and to build up a new economic sector.

But efforts to develop adoptive T cell therapies for solid tumors have hit upon a number of challenges; the only gene therapies to show significant benefit for patients have been in liquid tumors — forms of leukemia and lymphoma.

Supported by: Hear from leading expert Dr David DiGiusto, Stanford University School of Medicine, USA, as he shares his extensive experience in developing and optimizing manufacturing processes for cell and gene therapy products including:

Don't miss the chance to make new contacts, connect with current clients, develop your cell therapy, regenerative medicine and gene therapy business.

American Society of Gene and Cell Therapy Annual Meeting Speaker: Olivia Kelly, Ph.D., Senior Director, Cell Biology Presentation: Developing a Cell Therapy Combination Product for Diabetes Date / Time: May 16, 8:30 AM EDT Location: Hilton Chicago, Chicago

Oisín Biotechnology — a startup with initial funding from SENS Research Foundation, the Methuselah Foundation, and others — is close behind them, having developed a form of gene therapy that — in animals at least — also destroys these cells, using an approach that is less inherently likely to destroy healthy cells along with senescent ones.

Gene and cell therapies have made important medical advances over the past three decade, developing technologies and testing novel therapies in multiple human clinical trials of many diseases.