Differentiating hereditary from nonhereditary retinoblastoma is now possible overnight, improving early intervention ...

Oct. 28, 2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing function. These preclinical ...

The Mass General Brigham Gene and Cell Therapy Institute, a hub of innovation dedicated to accelerating groundbreaking research, conducting clinical trials and developing FDA-approved treatments, ...

Epilepsy is very common in tuberous sclerosis complex and occurs in 80 to 90% of affected individuals during their lifetime. Onset usually occurs during childhood, and up to one third of children ...

Using STC-1 cell line and different mouse models, including Neurog3-Tsc1−/− mice, rapamycin or L-lucine treatment to stimulate mTOR activity, we have demonstrated that mTOR stimulates proglucagon gene ...

CRISPR/Cas9 is a gene editing tool that has revolutionized biomedical research and led to the first FDA-approved CRISPR-based gene therapy. However, until now, the precise mechanism of exactly how ...

About Tuberous Sclerosis Complex Tuberous sclerosis complex (TSC) is a rare, multisystem genetic disorder caused by inherited mutations in the TSC1 gene or TSC2 gene. It is often characterized by ...