New Gene Therapy Approach Holds Potential Therapeutic Value in LAL-D
Researchers from France developed a gene therapy approach based on hematopoietic stem cells for the erythroid-specific expression of lysosomal acid lipase (LAL) enzyme in the blood as a potential curative treatment for LAL deficiency (LAL-D). The LAL enzyme is essential for the hydrolysis of cholesteryl ester and triglyceride in the lysosome. So its deficiency leads…