Tag: gene therapy

A new commentary explains how the tightly regulated, slowly renewing beta-cell mass of the pancreas creates critical therapeutic windows for ...

Researchers report that a novel combination of plasmids delivered directly into tumors induces high levels of regression in metastatic melanoma, ...

Researchers discovered a naturally nuclease-dead miniature CRISPR protein, engineered it into the hypercompact xCas12m, and showed that a single-AAV xCas12m–CRISPRoff ...

Kyoto University researchers used adenine base editing to correct the TFG mutation behind HMSN-P, delaying disease onset and extending survival ...

A new Gene Therapy article synthesizes evidence on how patients, consumers and societies value, perceive and prefer high-cost gene therapies ...

Researchers have fused HMGN1 and VP64 to a PAM-flexible, truncated-CDA1 base editor, boosting precision and efficiency for editing nearly any ...

Researchers engineered a cell-penetrating peptide, PF14-K, that delivers CRISPR ribonucleoproteins and DNA repair templates into primary human skin cells with ...

Researchers report a prime-editing strategy that integrates large DNA fragments into precise genomic sites through donor-directed annealing without double-strand breaks.

Researchers report that a technique called TRANsCre-DIONE can convert scar-forming reactive astrocytes into functional motor neurons, a result with implications ...

A new Gene Therapy review charts how mutation-agnostic optogenetic gene therapy could restore vision in blind patients, while detailing the ...