CRISPR-Cas systems are prokaryotic immune ... photoreceptors when delivered with adeno-associated viral gene therapy. SeqCas9, a Cas9 variant from the Streptococcus family, recognizes a simple ...
A temporally resolved CRISPR-Cas9 screen reveals DAC-induced DNA damage drives trans-cell cycle cytotoxicity that depends on ...
CRISPR technology has radically transformed genetic research, and at the forefront of this transformation is CRISPR screening ...
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One shot to lower cholesterol for life? Scientists unlock a groundbreaking gene therapyResearchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...
and recently added to its portfolio with a therapy for blood disorders sickle cell disease (SCD) and transfusion-dependent beta thalassaemia. It has broadened its R&D focus in recent years to ...
The drugmaker also won a CHMP endorsement for its CAR-T therapy Breyanzi ... get a speedy FDA review after the agency granted priority review to the company’s new drug application.
In multiple sclerosis (MS), a person's immune system attacks the protective covering (myelin sheath) of nerve fibers in the brain and spinal cord (central nervous system). MS disease-modifying ...
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