Sentences with phrase «human gene therapy»

A virus that has shown promise as a vector for human gene therapy causes liver tumors in neonatal mice.

The first successful targeted human gene therapy was reported in 2000.

Most recently, Dr. Gringeri was the Chief Operating Officer for Amsterdam Molecular Therapeutics (AMT), a Netherlands - based company engaged in human gene therapies for orphan diseases related to metabolic disorders, liver diseases, blood diseases, and disorders of the central and peripheral nervous systems.

«One of the potential issues with AAV is that there is no good way to control the expression of Cas9,» says Mark Kay, director of the Program in Human Gene Therapy at Stanford University.

«Those were very dark days when people worried the field was not going to survive,» says David T. Curiel, director of the division of human gene therapy at the University of Alabama at Birmingham.

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.

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.

The method, reported in the November issue of Nature Biotechnology, could lead to safe and effective human gene therapies for cystic fibrosis, hemophilia, and a variety of other diseases.

Field reports suggest that not all K13 mutations are capable of causing resistance, and the genetic system developed by Dr. Fidock to study K13, based on DNA repair approaches that are being used in human gene therapy studies, will be critical in identifying real hot spots of resistance.

Collins had been corresponding regularly with a leading human gene therapy proponent, James Wilson, who soon moved his lab to the University of Michigan, right next door to Collins» own.

They then stitched the genes for these immunoadhesins into an adeno - associated virus (AAV), a «vector» used in human gene therapy experiments to deliver foreign DNA into the body's cells.

Human Gene Therapy Clinical Development 25.3 (2014).

La Jolla, CA — New animal studies conducted at the Salk Institute for Biological Studies show that the only human gene therapy treatment to date...

A special issue of peer - reviewed journal Human Gene Therapy was published, documenting China's progress, opportunities and challenges in its biomedical research.

The research, part of a phase I clinical trial to test the safety of the treatment, was published as a letter to the editor in The New England Journal of Medicine earlier this week and will be in the September issue of Human Gene Therapy.

Theodore Friedmann, who directs the program in human gene therapy at the University of California at San Diego, spearheaded the first of these workshops six years ago.

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

In March 2002, Theodore Friedmann, who directs the program in human gene therapy at the University of California at San Diego and has advised the National Institutes of Health and congressional leaders on gene - related issues, organized a three - day workshop for the world agency.

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 place.

The author is director of the Program in Human Gene Therapy, University of California at San Diego School of Medicine, La Jolla, CA 92093 - 0634.

The former director of the Institute for Human Gene Therapy, he was widely regarded as the scientist most likely to crack the pesky vector problem: how to safely harness viruses as the Trojan horses that would carry new genes into human cells to repair their DNA.

To prevent this decline in Area X, White's research team used methods similar to human gene therapy to insert a version of FoxP2 in male zebra finches.

↵ * Present address: Department of Molecular and Cellular Engineering and Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA 19104, USA.

The FDA has also issued warning letters — a less severe sanction — to two of Wilson's collaborators in the study — Steven Raper of the Institute for Human Gene Therapy and Mark Batshaw of Children's National Medical Center in Washington, D.C.

For the past 3 months, faculty and staff members at the University of Pennsylvania's Institute for Human Gene Therapy have been trying to understand why a relatively fit 18 - year - old with an inherited enzyme deficiency died on 17 September, 4 days after doctors at Penn injected a genetically altered virus into his liver.

For applications in human gene therapy, improved methods for efficient and safe delivery of Cas9 / Cpf1 and their guide RNAs to cells and tissues are still needed.

«This is suggestive [of efficacy] and encourages us to move on,» says Crystal, whose team reports its results online today in Human Gene Therapy.

The findings also may prove helpful to researchers exploring pre-fertilization strategies for human gene therapy, thus allowing preventive treatment for certain genetic disorders, Dr. Burgess said.

Human Gene Therapy, 24 (12), A27 - A27.

Human Gene Therapy.

Approve of stem cell research, transgenic animal research and human gene therapy, although strict laws are needed to alleviate concern about ethical issues