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Genetics

Gene Therapy Gene Editing Obesity Treatment Rare Diseases Alzheimer's Disease Cardiomyopathy TERT Gene Expression BRAF Mutations Blood Disorders Breast Cancer Risk Assessment

Gene Therapy Restores Hearing in Most With OTOF Deafness, With Gains Lasting 2.5 Years

The multicenter trial shows the strongest gains in young children, signaling a path to clinical review in China.

Largest Study of Severe Pregnancy Sickness Confirms Key Gene and Finds Six New Leads

Noncoding U4/U6 RNA Variants Pinpointed as Cause of Retinitis Pigmentosa

Study Finds No Added Breast Cancer Risk From Menopausal Hormone Therapy in BRCA Carriers

Lurie Children’s Launches Genetics and Rare Diseases Division With $11 Million Edwards Gift

Genetic Risk Score Flags Future Breast Cancer Risk After DCIS or LCIS

Mount Sinai Team Publishes AI ‘Penetrance’ Scores to Gauge Disease Risk From Rare Variants

Pharmacogenetic Screening Halves Severe Chemotherapy Reactions in Gastrointestinal Cancer Patients

Largest Genetic Study Links Eight DNA Regions to ME/CFS

Global Genome Analysis Enables DNA-Based Stuttering Risk Prediction

Prime Editing Reverses Rare Childhood Brain Disorder by Correcting Multiple Mutations in Mice

Eight Healthy Babies Born Through UK Three-Parent IVF, Study Shows

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

CRISPR Technique Reawakens Dormant Genes to Treat Blood Disorders

Triplet Therapy Doubles Survival in BRAF V600E Metastatic Colorectal Cancer

Proteomic Test Cuts Childhood Rare Disease Diagnosis to Under Three Days

Comprehensive NGS Study Unveils Diverse Mutational Landscape in Lung Squamous Cell Carcinoma

New Genomic Score Enhances Prediction of Multiple Myeloma Progression

First In Vivo CRISPR Gene Therapy Saves Newborn with Rare Genetic Disorder

First Personalized CRISPR Therapy Corrects Rare Genetic Disorder in Infant