Remove DNA Remove Immune Response Remove Therapies
article thumbnail

Scientists engineer CRISPR enzymes that evade the immune system

Broad Institute

Scientists engineer CRISPR enzymes that evade the immune system By Allessandra DiCorato January 9, 2025 Breadcrumb Home Scientists engineer CRISPR enzymes that evade the immune system The new genome-editing tools could lead to safer, more efficient gene therapies.

article thumbnail

A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

Disease 142
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Scaling Phage Therapy

Codon

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

Therapies 122
article thumbnail

FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

Therapies 103
article thumbnail

Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

article thumbnail

Cell and gene therapy development moves into cardiac indications

Drug Target Review

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

article thumbnail

Not all neoantigens are created equal

Drug Target Review

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. Neoantigens are recognised as non-self and trigger an immune response.