Sentences with phrase «of human gene therapy»

Division of Human Gene Therapy, Department of Medicine, Department of Pathology, Department of Surgery, and the Gene Therapy Center, Birmingham, Alabama, UK

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.

«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.

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.»

They include going after the damage to cells done by free radicals, making use of hormone therapy, or caloric restrictions, or vitamin supplements, or, most dramatically, healthy gene selection through pre-implantation genetic diagnosis and even repairing the entire human genome.

The principles that have emerged thus far are these: We should seek new knowledge of our genes (and we can say this without deciding whether the Human Genome Initiative is the wisest and most cost - effective way to do so) We should seek therapies for the genetic disorders that afflict many people.

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.

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.

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.

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.

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 testing.

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.

The SMA model pig was created using a gene therapy approach by knocking down the levels of pig SMN, followed by treatment with human SMN at early and late time points.

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.

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.

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.

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.

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.

Gene therapy can be used to neutralise harmful human genes, such as those that cause cancer, or to insert healthy copies of damaged genes into people with a genetic disease.

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 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.

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.

If successful, it could eventually pave the way for gene - therapy tests of humans with MS.

Most gene - therapy trials use viruses to deliver genes to a patient's cells, and most of those viruses are retroviruses, which have the ability to neatly splice their genes — and the human gene they're carrying — into a cell's chromosomes.

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.

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

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

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.

The blood sugar of the diabetic mice were made normal by the gene - therapy - treated human islets on the right.

One of the most promising avenues for developing a cure, however, is through gene therapy, and to create those therapies requires animal models of disease that closely replicate the human condition.

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.

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.

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.

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.

This conference touched on a broad spectrum of topics encompassing scientific integrity, including gene therapy, guidelines for animal and human subject use, authorship, public health issues, and the involvement of minorities in research.

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.

Gene therapy has been rhapsodized and vilified in its nearly two decades of human testing, helping some and making others sicker.

«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 results, published in the current issue of Human Molecular Genetics, open the door for pursuing gene editing in nonhuman primates as models for new therapies, including pharmacological, gene - and stem cell - based therapies, said Keith Latham, MSU animal science professor and lead author of the study.