Single-dose gene therapy is potentially life-changing for adults with hemophilia B
Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71% after a single infusion of gene ...
News Medical2h
CRISPR-Cas13 emerges as a game changer in RNA-targeted therapies
In recent years, the scientific community has made significant strides in the field of gene editing, particularly through the ...
US FDA approves IntraBio's drug for rare genetic disease
Niemann-Pick disease type C (NPC) is a rare genetic disorder that affects the nervous system and other organs, causing ...
Nature21h
Children with Down's syndrome are more likely to get leukaemia: stem-cells hint at why
Children with Down’s syndrome have a 150-fold increased risk of developing leukaemia than those without the condition. Now, ...
WebMD1d
FDA OKs First Treatment for Rare, Deadly Genetic Disorder
Miplyffa treats Niemann-Pick disease type C, which progressively impacts the abilities to speak, swallow, and walk or move ...
Researchers identify genetic mutations for rare disorder causing cognitive decline
A rare cause of hereditary cognitive decline known as CSF1R-Related Disorder (CSF1R-RD) gets its name from mutations in the ...
Teen boy first in Michigan to get gene therapy treatment for blood disorder
Children's Hospital of Michigan is treating the first person in Michigan with a newly approved gene therapy for beta ...
News Medical1d
Known genes account for most undiagnosed cases of developmental disorders
Scientists have conducted the largest and most diverse study to date on how recessive genetic changes contribute to developmental disorders. They found that most undiagnosed cases that are due to ...
Nature1d
What is a cell type, really? The quest to categorize life’s myriad forms
“A cell type is a group of cells that are similar to each other and distinct from other groups of cells,” offers ...