Sentences with phrase «in human gene therapy»

The goal of the Program in Human Gene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseases.

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.

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

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.

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.

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

No cases of severe pancreatitis and only one admission to the intensive care unit for an LPLD - related abdominal event were reported in the study published in 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.

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.

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.»

Gene therapy procedures in humans have been linked to the onset of leukemia and various tumors, as well as sudden death.

Gene therapy delivered to a specific part of the brain reverses symptoms of depression in a mouse model of the disease — potentially laying the groundwork for a new approach to treating severe cases of human depression in which drugs are ineffective.

The scope of bioethics can expand with biotechnology, including cloning, gene therapy, life extension, human genetic engineering, astroethics and life in space, and manipulation of basic biology through altered DNA, XNA and proteins.

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.

The investigators caution the approach is years away from use in humans, but gene therapy carries the promise of restoring hearing in people with several forms of both genetic and acquired deafness.

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.

So far, gene therapy attempts have only resulted in partial improvements of hearing in mouse models of specific human deafness forms that did not include severe anomalies in hair cell structure.

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.

This is advantageous, since dogs provide new models to investigate the disease mechanisms and to plan new therapies such as gene therapy, successfully applied to blindness in dogs and human previously,» explains Lohi.

Human testing is years away, but gene therapy has already become a controversy in professional and amateur sports, where steroids, human growth hormone, and other performance - enhancing drugs have been a problem for yHuman testing is years away, but gene therapy has already become a controversy in professional and amateur sports, where steroids, human growth hormone, and other performance - enhancing drugs have been a problem for yhuman growth hormone, and other performance - enhancing drugs have been a problem for years.

A team of researchers at the Stanford University School of Medicine has used a gene - editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.

More and more, Sweeney says, the immune system is proving to be the most difficult hurdle in developing gene therapy for humans.

In the 1990s scientists such as himself, he explains, were too caught up in the promise of gene therapy to realize that they did not know enough about it to warrant human testinIn the 1990s scientists such as himself, he explains, were too caught up in the promise of gene therapy to realize that they did not know enough about it to warrant human testinin the promise of gene therapy to realize that they did not know enough about it to warrant human testing.

But to do the gene therapy in humans, scientists would need to tackle another problem.

«If this approach works in humans, it will really change the conversation that providers have with patients,» Scadden said, especially for those «who have these underlying genetic disorders and for who the new gene - editing and gene therapy techniques are being developed.»

One clinical trial involves the drug CGF166, a one - time gene therapy, which, if proven successful in humans, could regenerate new hair cells within the cochlea that can signal the part of the brain that processes sound.

Before moving on to human trials, they will need to study all instances of «off - target» effects: Years before Crispr, the viruses employed to deliver DNA in gene therapy trials occasionally damaged the whole system, causing cancer.

The lack of discrimination in the viruses used to carry therapeutic genes has been a major obstacle to testing gene therapy in humans.

Skeletal muscle is one of the largest tissues in the human body and current gene therapy methods are only able to affect a portion of the muscle.

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.

«Another future goal, however, is to use CRISPR - Cas9 for somatic gene therapy in humans with severe diseases,» Klaus Rajewsky pointed out.

Still, by identifying the human hairless gene as an important master switch in regulating cell death in a hair follicle — a discovery that could lead to gene therapies for unwanted hair growth — Christiano emerged as a new star in the field, and a glamorous one.

29 GENETICALLY MODIFIED SUPERHUMANS The debate over human germ - line engineering — reworking genes in the sperm and egg to create inheritable new traits — sputtered out early in the last decade after gene therapy had a series of notable failures.

In humans, variants of the IL - 15R - alpha gene have been found in world - class endurance competitors, suggesting a target for gene therapies aimed at boosting the ability to exercise longeIn humans, variants of the IL - 15R - alpha gene have been found in world - class endurance competitors, suggesting a target for gene therapies aimed at boosting the ability to exercise longein world - class endurance competitors, suggesting a target for gene therapies aimed at boosting the ability to exercise longer.

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.

Human testing is years away, but gene therapy has already become a controversy in professional and amateur sports, where steroids, human growth hormones, and other performance - enhancing drugs have been a problem for yHuman testing is years away, but gene therapy has already become a controversy in professional and amateur sports, where steroids, human growth hormones, and other performance - enhancing drugs have been a problem for yhuman growth hormones, and other performance - enhancing drugs have been a problem for years.

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

Although the gene / cell therapy strategy was highly successful in laboratory mice, the authors stressed that additional research and testing are needed before the therapy could be tested in humans.

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.

New studies could include gene therapies in humans with visual problems like macular degeneration or retinal cancer.

Penn Vet researchers have had success in treating various forms of blindness in dogs with gene therapy, setting the stage to treat human blindness.

The light - activated genetic switch could be used to turn genes on and off in gene therapies; to turn off gene expression in future cancer therapies; and to help track and understand gene function in specific locations in the human body.

The roadmap outlines future research directions toward the goal of enhancing human radioresistance, including upregulation of endogenous repair and radioprotective mechanisms, possible leeways into gene therapy in order to enhance radioresistance via the translation of exogenous and engineered DNA repair and radioprotective mechanisms, the substitution of organic molecules with fortified isoforms, the coordination of regenerative and ablative technologies, and methods of slowing metabolic activity while preserving cognitive function.

It uses a virus already approved by the Food & Drug Administration for other genetic therapies in the eye; it delivers an ion channel gene similar to one normally found in humans, unlike others that employ genes from other species; and it can easily be reversed or adjusted by supplying new chemical photoswitches.

The specificity of this DNA cutting activity has made CRISPR - Cas the darling of gene therapy researchers, who have modified it to make precise changes in the genomes of cultured cells, laboratory animals, and even humans.

The team at UF's Powell Center for Rare Disease Research and Therapy conducted the first in - human study of gene therapy to treat respiratory dysfunction in patients with infantile onsetTherapy conducted the first in - human study of gene therapy to treat respiratory dysfunction in patients with infantile onsettherapy to treat respiratory dysfunction in patients with infantile onset Pompe.

For the animal experiments, Savio Woo of the Center for Gene Therapy at Baylor College of Medicine in Houston and his colleagues first isolated liver cells from transgenic mice that produce the human protein a1 - antitrypsin in their livers, from where it is secreted into the blood.

Mind controls: Putting a light switch in the brain With optogenetics, researchers can implant optical fibres to control genetically modified animals — could gene therapy bring it to humans?