English
Arabic
Chinese (Simplified)
Dutch
Esperanto
French
German
Greek
Italian
Japanese
Korean
Portuguese
Russian
Spanish
UrduAdvances in gene therapy may aid future treatments of Alzheimer’s disease, Parkinson’s disease, and more
Tag: Gene Therapy
Researchers quantify Cas9-caused off-target mutagenesis in mice
Findings reported at ASHG 2019 Annual Meeting
NIH funds new All of Us Research Program genome center to test advanced sequencing tools
HudsonAlpha awarded $7 million to expand national health dataset with uncharted genetic variants
New research center to explore muscle health and disease
With collaborating labs across the University of Washington campus and at other Seattle-area institutions and beyond, the Center for Translational Muscle Research will encompass a myriad of muscle science and disease investigations.
Researchers Develop mouse model of human gene involved in Alzheimer’s disease
Findings reported at ASHG 2019 Annual Meeting
Researchers glean new insights into biological underpinnings of schizophrenia
Findings reported at ASHG 2019 Annual Meeting
In-office gene therapy for wet age-related macular degeneration is coming
New data shows patients maintaining vision after a single intravitreal injection of gene therapy
Genetics of nephropathia epidemica researched by Kazan Federal University
A paper appeared recently in Viruses
New research uncovers how common genetic mutation drives cancer
Findings in Nature show that targeted therapy for mutated gene can slow or prevent tumor growth in many kinds of cancer
Carnegie Mellon and Yale receive NIH grant to further gene editing technique
The peptide nucleic acid-based technique offers an alternative to CRISPR-Cas9
Stem cell studies offer hope for childhood neurological condition
As both therapy and tool for drug discovery, stem cells present parallel opportunities for treatment
Identifying a gene for canine night blindness
An international team including University of Pennsylvania researchers has identified the mutation associated with a form of canine night blindness that also affects humans
ASHG 2019 in Houston highlights discoveries in genetic research and progress
Improve health, treat disease
Breakthrough in sex-chromosome regulation
Researchers at Karolinska Institutet in Sweden have uncovered a chromosome-wide mechanism that keeps the gene expression of sex chromosomes in balance in our cells. The findings shed light on molecular reasons for early miscarriage and could be important for the…
Grant will test ‘home delivery’ for CRISPR cancer treatment
What if, instead of killing cancer cells or cells about to become cancerous, you could deliver a repair package and fix them? UC Davis researchers have just been awarded $1.5 million from the National Institutes of Health (NIH) to start…
NIH researchers create new viral vector for improved gene therapy in sickle cell disease
‘Forward-oriented’ design might boost treatment effectiveness and broaden use
Johns Hopkins Researchers Advance Search For Safer, Easier Way to Deliver Vision-Saving Gene Therapy to The Retina
In experiments with rats, pigs and monkeys, Johns Hopkins Medicine researchers have developed a way to deliver sight-saving gene therapy to the retina. If proved safe and effective in humans, the technique could provide a new, more permanent therapeutic option for patients with common diseases such as wet age-related macular degeneration (AMD), and it could potentially replace defective genes in patients with inherited retinal disease.
New CRISPR-Cas9 variant may boost precision in gene editing
Researchers have developed a new variant of the gene editing technique CRISPR-Cas9 that has the potential to increase precision during gene therapy in humans. The new variant reduced unintended changes in DNA compared to its wildtype, suggesting it could play…
Genetic markers linked to the start of symptoms of Parkinson’s disease
New therapeutic targets
7-year-old receives FDA-approved gene therapy
University of Michigan Kellogg Eye Center is helping to save a child’s eyesight with gene therapy.
New CRISPR class expands genetic engineering toolbox
The new approach opens up nearly 90 percent of CRISPR-Cas systems for use in human cells, including biomedical research and potential gene and cell therapies
Stem cell researchers reactivate ‘back-up genes’ in the lab
Researchers in Belgium present new findings on X chromosome reactivation
Some cancer drugs in clinical trials don’t work by hitting their targets
Multiple cancer drug candidates in clinical trials kill tumor cells through off-target effects instead of by interacting with their intended molecular targets, according to a new study. The unexpected findings demonstrate that the targets of these drugs are not essential…
Trial seeks to reduce neuropathy, improve outcomes for black women with breast cancer
Researchers at Indiana University School of Medicine have launched a new, unique study recruiting black women with breast cancer designed to better understand and treat neuropathy, a side effect from chemotherapy. The National Cancer Institute-sponsored clinical trial, EAZ171, is led…
NSF Awards ‘Dream Team’ $1.1 Million for Blind Mexican Cavefish Research
How is it that a fish that lived in difficult to access caves in rural Mexico is becoming a leading model to study diabetes, insomnia, and obesity? It’s all about this tiny, translucent, cave-dwelling fish that lost its eyes to…
National Institutes of Health director hails SUNY Downstate for vision research
Neurotechnology pathway could help end blindness for people with age-related macular degeneration (A
National Institutes of Health director hails SUNY Downstate for vision research
Neurotechnology pathway could help end blindness for people with age-related macular degeneration (A
A novel technology for genome-editing a broad range of mutations in live organisms
Salk scientists develop a new gene-editing tool that could help treat many disorders caused by gene
Tweaked CRISPR in neurons gives scientists new power to probe brain diseases
A team of scientists at UC San Francisco and the National Institutes of Health have achieved another CRISPR first, one which may fundamentally alter the way scientists study brain diseases. In a paper published August 15 in the journal Neuron…
AAV9 gene therapy vector dramatically increases life span in krabbe disease mouse model
New Rochelle, NY, August 12, 2019–An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe dis-ease improved…
NIH’s All of Us Research Program recaps progress and next steps
WHAT: The All of Us Research Program at the National Institutes of Health has made strong progress in its efforts to advance precision medicine, according to program leadership in a forthcoming paper in the New England Journal of Medicine .…