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gene therapy

Model of PEDF protein alongside the 17-mer and H105A peptides. Amino acid 105, which is changed from histidine in PEDF and the 17-mer peptide to alanine in the H105A peptide, is shown in green.

NIH researchers develop eye drops that slow vision loss in animals

Categories Health, Life & Non-humans

sickle cells

New Transplant Method Shows Promise as Accessible Cure for Sickle Cell Disease

Categories Health

Jace with his father Brendan

Genetic therapy gives infants life-changing improvements in sight

Categories Health

Cryo-EM analysis of designed de novo protein nanocages

AI Designs Complex Protein Structures Inspired by Viral Architecture

Categories Health

Light-induced gene therapy disables cancer cells’ energy center

Categories Technology

Robin Shaw, MD, PhD (left) and TingTing Hong, MD, PhD (right) at the lab bench.

New Gene Therapy Shows Unprecedented Heart Failure Recovery in Large Animal Study

Categories Health

The CRISPR gene scissors can correct mutations in DNA very precisely. However, the treatment of genetic defects can lead to new errors in the genome that may pose health risks. (Image: iStock/luismmolina)

Scientists Uncover Hidden Risks in CRISPR Gene Editing for Rare Immune Disease

Categories Health, Technology

Michael is checked by a nurse at SickKids before receiving gene therapy in a single-patient clinical trial for spastic paraplegia type 50 (SPG50).

Gene therapy halts progression of rare genetic condition in young boy

Categories Health

By making grafts called cultured epidermal autografts (photo), which contain genetic mutation corrections that give healthy skin, and grafting these naturally corrected skin cells to affected areas, outbreaks of the disease could be controlled.

Treating rare skin diseases by transplanting healthy skin

Categories Health

Julia Aguade Gorgorio (left) and Dr. Hanna Mikkola (right)

Scientists identify ‘missing piece’ required for blood stem cell self-renewal

Categories Health

Foam containing gene therapy components is placed in a lab dish.

Medicated foam could make gene therapies more accessible

Categories Health, Technology

Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

Engineered Virus Crosses Blood-Brain Barrier, Delivers Therapeutic Genes in Mice

Categories Brain & Behavior

Gene therapy relieves back pain, repairs damaged disc in mice

Categories Technology

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