Retrograde AAV selectively targets D1-MSNs and rescues Parkinsonian symptoms with chemogenetic modulation. Credit: SIAT Scientists from the Shenzhen Institute of Advanced Technology (SIAT) within the…
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Whole spinal cord visualization of regenerating projections from the lower thoracic spinal cord that project to walking execution centers. Credit: EPFL / .Neurorestore Guiding cells…
The new tool has the potential to improve gene and cell therapies. Scientists at St. Jude Children’s Research Hospital have developed a tool that can…
The patients had some, although severely diminished, visual function during the day, however, at night they were essentially blind, with light sensitivity 10,000–100,000 times less…
Researchers discovered two compounds that are more potent and less toxic than existing leukemia treatments, which often cause unpleasant side effects in patients. Researchers discover…
Could gene therapy cure colorblindness in the future? Following treatment, two of the four children’s measurements of cone function closely resembled those of normal-sighted research…
Although CRISPR therapeutics are incredibly effective, the researchers warn that they could promote cancer. The study showed that CRISPR therapeutics can damage the genome. The…
Depression is a widespread and serious medical condition that has a negative impact on how you feel, think, and behave. Positive results from a study…
To identify the genes, the researchers used data from 723,266 persons from 17 studies who had clinically diagnosed or self-reported hearing impairment and examined genetic…
A new study has shown that gene therapy may be able to prevent or reverse many deleterious effects of Pitt-Hopkins syndrome New research from the…
Researchers have discovered a new way in which cancer cells can repair DNA damage. Identifying DNA Repair Genes Holds Promise for Improving Cancer Treatment Cancer…
A new gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain in mice with spinal cord or peripheral nerve injuries with no…
Findings could lead to targeted approach for treating aging. Research from the Babraham Institute has developed a method to ‘time jump’ human skin cells by…
This cross-section of a mouse heart (red) shows how well the gene therapy delivered sodium ion channel genes (cyan) to the target heart cells after…
A collaborative research team at Harvard’s Wyss Institute and the ETH Zurich in Switzerland has identified genomic safe harbors (GSHs) in the tumultuous sea of…
Novel obesity treatments such as modulation of the gut microbiome and gene therapy are underutilized and could help fight the obesity epidemic, according to a…
Researchers Engineer a Mini CRISPR Genetic Editing System That Could Be Easier To Deliver Into Cells
Researchers Engineer a Mini CRISPR Genetic Editing System That Could Be Easier To Deliver Into Cells
CRISPR illustration. Credit: National Institutes of Health A compact and efficient CRISPR-Cas system, named CasMINI, could be broadly useful for cell-engineering and gene-therapy applications because…
Stanford researchers have engineered a new miniature CRISPR system that should be easier to deliver into human cells. Bioengineers have repurposed a “non-working” CRISPR system…
Mount Sinai researchers have developed a therapeutic agent that shows high effectiveness in vitro at disrupting a biological pathway that helps cancer survive, according to…
Staining for RAD51 (bright cyan-colored dot) in a fertilized one-cell mouse embryo shows repair of a CRISPR-induced DNA break. Credit: Image courtesy of the researchers.…