Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

TSRI scientists find new way to make cell population resistant to HIV

Scientists at The Scripps Research Institute have found a way to tether HIV-fighting antibodies to immune cells, creating a cell population resistant to the virus.

11 Apr 2017

Researchers use genetic manipulation to regenerate auditory hair cells in adult mice

In an apparent first, St. Jude Children's Research Hospital investigators have used genetic manipulation to regenerate auditory hair cells in adult mice. The research marks a possible advance in treatment of hearing loss in humans. The study appears today in the journal Cell Reports.

11 Apr 2017

TSRI professor receives $4.8 million grant to advance potential HIV vaccine research

Professor Michael Farzan, co-chair of the Department of Immunology and Microbiology on the Florida campus of The Scripps Research Institute has received $4.8 million in funding through a 2017 Avant-Garde Award for HIV/AIDS research from the National Institutes of Health's National Institute on Drug Abuse.

11 Apr 2017

Scientists use time-lapse video to identify cells that trigger blood stem cell development

Like private investigators on a stake out, St. Jude Children's Research Hospital scientists used patience and video surveillance-like tools to identify cells that trigger blood cell development.

10 Apr 2017

Researchers use modified AAV as ‘Trojan Horse’ for delivering genes to restore congenital deafness

There are more than 300 genetic defects that have been found to prevent the hair cells in the human inner ear, the sensory cells of the ear as it were, from working properly.

6 Apr 2017

ISCT honors Dr. Adrian Gee with career achievement award

The International Society for Cellular Therapy, the global society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell therapy sector, today announces that Dr. Adrian Gee, Professor of Medicine and Pediatrics at the Baylor College of Medicine in Texas, US, and Director of the Clinical Applications Laboratory at the Center for Cell and Gene Therapy, is to be awarded the 2017 ‘ISCT Career Achievement Award in Cellular Therapy’.

6 Apr 2017

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy.

5 Apr 2017

Thermo Fisher Scientific collaborates with CGT Catapult to create seamless cell and gene therapy supply chain

To help address the challenges surrounding supply chain management for cell and gene therapy around the globe, Thermo Fisher Scientific, the world leader in serving science, today announced a collaboration with the Cell and Gene Therapy Catapult to provide developers with both the manufacturing capability and distribution, logistics, and storage capacity needed to create a seamless supply chain to accelerate cell and gene therapy development and commercialsation.

From Thermo Fisher Scientific Inc. 24 Mar 2017

Inner ear gene therapy holds promise for treating patients with hearing loss and dizziness

In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders and Johns Hopkins University School of Medicine showed that gene therapy was able to restore balance and hearing in genetically modified mice that mimic Usher Syndrome, a genetic condition in humans characterized by partial or total hearing loss, dizziness, and vision loss that worsens over time.

23 Mar 2017

Efficacy of innovative gene therapy can be tested quickly and cost-effectively using new cellular model

Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy.

21 Mar 2017

Innovative strategy uses genetically modified viruses to selectively attack tumor cells

Scientists at the IDIBAPS Biomedical Research Institute and at the Institute for Research in Biomedicine lead a study in which they have designed a new strategy to get genetically modified viruses to selectively attack tumor cells without affecting healthy tissues.

16 Mar 2017

Study findings could lead to novel therapies for preventing vision loss from degenerative retinal diseases

Silencing a gene called Nrl in mice prevents the loss of cells from degenerative diseases of the retina, according to a new study.

15 Mar 2017

Researchers discover new way to curb chronic pain

For millions of sufferers, there is nothing more debilitating than chronic back or joint pain. It can feel like a lifetime of misery.

14 Mar 2017

Study identifies protein that may offer protection against heart disease

A protein found in the heart that is known to be involved in cellular stress responses in cancer cells is now believed to play a critical role in the ability of cardiac cells to combat heart disease and recover from a heart attack.

6 Mar 2017

Study describes method to engineer brown adipose tissue from white fat-derived stem cells

Researchers have demonstrated the potential to engineer brown adipose tissue, which has therapeutic promise to treat metabolic diseases such as obesity and type 2 diabetes, from white adipose-derived stem cells.

3 Mar 2017

Innovative gene therapy allows complete remission of clinical signs of sickle cell disease

A team led by Pr. Marina Cavazzana conducted at Necker Children's Hospital, AP-HP and the Imagine Institute in October 2014 gene therapy in the context of a phase I clinical trial / II in a patient 13 years with severe sickle cell disease.

2 Mar 2017

Cedars-Sinai researchers move closer to developing biological pacemaker with new $3 million NIH grant

With a new $3 million grant from the National Institutes of Health, Cedars-Sinai Heart Institute investigators are moving closer to their goal of developing a biological pacemaker that can treat patients afflicted with slow heartbeats.

21 Feb 2017

Scientists engineer smallest CRISPR-Cas9 system to date

Scientists at the Center for Genome Engineering, within the Institute for Basic Science0, in collaboration with KIM Eunji and KIM Jeong Hun have engineered the smallest CRISPR-Cas9 to date, delivered it to the muscle cells and in the eyes of mice via adeno-associated viruses and used it to modify a gene causing blindness.

21 Feb 2017

Glowing mice could mark major step forward for new gene therapy

Timothy Blake, a postdoctoral fellow in the Waymouth lab, was hard at work on a fantastical interdisciplinary experiment. He and his fellow researchers were refining compounds that would carry instructions for assembling the protein that makes fireflies light up and deliver them into the cells of an anesthetized mouse. If their technique worked, the mouse would glow in the dark.

16 Feb 2017

Canadian researchers use gene therapy to treat patient with Fabry disease

A team of Canadian physicians and researchers is believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten lifespan.

16 Feb 2017

Gene Therapy News and Research

Further Reading

TSRI scientists find new way to make cell population resistant to HIV

Researchers use genetic manipulation to regenerate auditory hair cells in adult mice

TSRI professor receives $4.8 million grant to advance potential HIV vaccine research

Scientists use time-lapse video to identify cells that trigger blood stem cell development

Researchers use modified AAV as ‘Trojan Horse’ for delivering genes to restore congenital deafness

ISCT honors Dr. Adrian Gee with career achievement award

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

Thermo Fisher Scientific collaborates with CGT Catapult to create seamless cell and gene therapy supply chain

Inner ear gene therapy holds promise for treating patients with hearing loss and dizziness

Efficacy of innovative gene therapy can be tested quickly and cost-effectively using new cellular model

Innovative strategy uses genetically modified viruses to selectively attack tumor cells

Study findings could lead to novel therapies for preventing vision loss from degenerative retinal diseases

Researchers discover new way to curb chronic pain

Study identifies protein that may offer protection against heart disease

Study describes method to engineer brown adipose tissue from white fat-derived stem cells

Innovative gene therapy allows complete remission of clinical signs of sickle cell disease

Cedars-Sinai researchers move closer to developing biological pacemaker with new $3 million NIH grant

Scientists engineer smallest CRISPR-Cas9 system to date

Glowing mice could mark major step forward for new gene therapy

Canadian researchers use gene therapy to treat patient with Fabry disease

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