Collaborate Disseminate
A long-term follow-up study tracking a group of children who received experimental gene therapy as part of an early-stage clinical trial is reporting the treatment is safe and effective up to 10 years later. The research offers rare insight into the lo… Continue reading One-off gene therapy for rare immune disease still effective 10 years later
One of the most revolutionary discoveries of the century, the CRISPR gene-editing system is built on a bacterial defense mechanism that allows scientists to make precise edits to DNA. Now scientists at MIT have discovered a new class of enzymes called … Continue reading Beyond CRISPR: New class of gene-editing enzymes discovered
The CRISPR gene-editing system is usually associated with the Cas9 protein, but that’s not the only option out there. Scientists at Stanford have now developed a CRISPR tool called CasMINI, using a much smaller protein that should be able to enter huma… Continue reading “Mini” CRISPR tool reworks inactive protein for better gene editing
New data from the United States’ first CRISPR gene editing clinical trial indicates the treatment is safe and effective more than two years after initial dosing. Targeting a pair of rare genetic blood diseases, the experimental therapy has so far been … Continue reading CRISPR therapy for rare blood disease delivers “life-changing” results
A landmark study published in the journal Nature Communications is describing the extraordinarily successful results of a Phase 1 trial testing a targeted gene therapy for children with a rare genetic disease. The research demonstrates a novel method f… Continue reading Gene therapy success offers hope for reversing rare genetic diseases
Scientists are reporting the first clinical data showing that CRISPR gene editing can be done safely and effectively inside the body. CRISPR was injected directly into the bloodstream of patients with a rare genetic disease, and appeared to work better… Continue reading CRISPR used to treat rare genetic disease in promising phase 1 trial
An experimental gene therapy developed to treat children born with a rare immunodeficiency disease has demonstrated extraordinary efficacy according to a new long-term follow-up study published in the New England Journal of Medicine.Continue ReadingCat… Continue reading Experimental gene therapy points to cure for rare immune disease
CRISPR-Cas9 is a revolutionary gene-editing tool, but it’s not without its downsides. Now, scientists at Harvard have demonstrated an alternative genetic engineering system called Retron Library Recombineering (RLR), which works without cutting DNA and… Continue reading Harvard gene-editing tool “sneaks” DNA into cells without making cuts
There are a lot of unknowns when it comes to the way Alzheimer’s takes hold in the brain, but one area where researchers are focusing much of their attention is the development of toxic clumps of proteins in the brain. These harmful clusters are seen as key players in the cognitive decline associated with the disease, and scientists have now found that a single injection of a novel gene therapy may be able to prevent a significant portion of them from forming.
Tags: Alzheimer’s Disease, Dementia, Massachusetts General Hospital, Genetics, gene therapy, New Atlas Audio
Continue reading “Zinc finger” gene therapy tackles the toxic tangles of Alzheimer’s
An intriguing new proof-of-concept study from a team of University of California San Diego researchers suggests CRISPR gene therapy may be a promising alternative to opioids for chronic pain. The preliminary investigation demonstrated lowered pain sens… Continue reading Unique CRISPR gene therapy offers opioid-free chronic pain treatment