Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.
Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...
CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...
Global clinical-stage biotech company HuidaGene Therapeutics has received FDA clearance to move forward with its application for its investigational drug, a CRISPR/Cas13 RNA-editing therapy to treat ...
Like the human immune system, bacteria learn from past infections. CRISPR sequences—short snippets of DNA from previous viruses—guide destructive enzymes towards invading bacteriophages that express ...
For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby ...
Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...
A powerful form of DNA-editing machinery discovered in bacteria might allow us to make much bigger changes to genomes than is currently possible with CRISPR-based techniques. However, it isn’t yet ...
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