Mapping mutations at scale in a single gene reveals new neurodevelopmental condition

The ability of different genetic variants—changes to one or more building blocks of DNA—to cause disease, and to what extent, ...
Labroots

This Crucial Gene Activator Can Also Function as a Repressor

During development, many crucial genes help organisms form properly. One of those genes is myogenic determination gene number 1 (MyoD), which can exert a powerful influence as a “genome organizer." It ...
GEN

Gene Therapy Reverses Effects of Heart Failure and Restores Heart Function in Minipigs

Researchers at the Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah, and the University of Utah School of Medicine, have demonstrated that a gene therapy can ...

UCLA-led gene therapy clinical trial leads to FDA approval for rare immune disorder

A groundbreaking gene therapy tested through a UCLA-led clinical trial has received approval from the U.S. Food and Drug ...
GEN

GeneAgent Reduces AI Hallucinations in Gene Function Prediction

Gene-set analysis seeks to identify the biological mechanisms underlying groups of genes with shared functions. Large language models (LLMs) have shown promise in generating functional descriptions ...
HUB

New AI tool pinpoints gene splicing with unmatched precision

Johns Hopkins researchers have developed a powerful new AI tool called Splam that can identify where splicing occurs in genes—an advance that could help scientists analyze genetic data with greater ...
Phys.org

Genome editing technique enables partial inhibition of gene function in crops

Researchers at Kumamoto University have successfully executed a practical trial of a new genome-editing technique that allows for the partial inhibition of essential gene function without causing ...
Ophthalmology Times

FDA grants RMAT designation to RTx-015 optogenetic gene therapy for retinitis pigmentosa

RMAT designation enables intensified FDA engagement and potential accelerated pathways, implying preliminary clinical evidence for RTx-015 despite absent peer-reviewed efficacy details, endpoints, and ...
Healio

Gene therapy improves motor function in spinal muscular atrophy at 52 weeks

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Patients treated with onasemnogene abeparvovec recorded ...
News Medical

Unexpected function of gene network in gut microbes revealed

Could a gene regulatory network in gut microbes have evolved its elaborate and tightly regulated molecular machinery only to pump out antibiotics indiscriminately? Researchers from the Institute of ...
Medscape

Gene Therapy Shows Signal for Improving Retinal Function

A gene therapy that targets a form of retinitis pigmentosa showed a signal to improve retinal function for up to 3 years after treatment, according to a small clinical trial. The phase 2 SKYLINE trial ...