Raymond Wang, MD

Children’s Hospital of Orange County (CHOC)

Translational research for next generation treatments and cures for lysosomal storage disorders

Raymond Wang, MD is the Director of the Multidisciplinary Lysosomal Storage Disorder Program at the Children’s Hospital of Orange Country (CHOC) and a board certified clinical geneticist and biochemical genetics specialist.    

Dr Wang conducts basic scientific research into lysosomal storage disorders (LSD’s) including Salla Disease.  Dr. Wang’s team is particularly interested in the development of new treatments that can access more challenging parts of the body, such as the brain, especially important in the case of Salla.

Raymond Wang next to fellow researchers wearing lab coats.

Dr Wang also maintains an active clinical practice treating patients and evaluating new therapeutic interventions for currently untreatable lysosomal storage disorders.  

A group of people standing in front of a white board.

CRISPR gene editing holds potential in the potential treatment of genetic disorders such as Salla because the underlying genetic alterations can be directly addressed.  CRISPR proteins act like a pair of precision scissors and thread.  Scientists can give cells the correct sequence to use for repair templates, correcting the disease-causing DNA sequences.

An illustration showing scissors cutting DNA that has a harmful gene variant. The DNA is then repaired, restoring a healthy variant.

Dr Wang’s lab is pioneering the application of CRISPR approaches to cells that contain the common p.R39C variant with a view to demonstrating that sialic acid transport can be corrected through the expression of an edited copy of the SLC17A5 gene.  

Dr Wang’s team has the expertise and infrastructure to then translate promising research findings into therapeutic applications of the CRISPR technology for patients with Salla disease.  

As with the development of any new medicine, many technical hurdles will need to be overcome along the way including how to deliver the CRISPR proteins effectively and safely to the brain.

Dr Wang is a member of the STAR Foundation’s Scientific Advisory Board where his deep experience of basic science and translational medicine can be fully leveraged.