ASO TREATMENT

Antisense Oligonucleotides (ASO) Treatment: This treatment would reduce the expected toxicity to cells from the mutated tubulin by using an approach that captures the mutated RNA before it becomes a damaged protein. Unlike the AAV treatment, ASO treatment is not a permanent cure and requires ongoing distribution of the serum to the patient to maintain the reduction of the toxicity. Therefore, while we wait for a permanent cure, the ASO will extend the lives of affected patients. Read more about ASOs in Nature: here.

Principal Investigator

A team of researchers at Children’s Hospital of Philadelphia (CHOP) led by Dr. Adeline Vanderver is now at the interim stage of their research for a cure using ASOs. Current results indicate they are on the right path toward launching a clinical trial in the near future. 

CHOP TEAM

“In the last decade there’s really been revolutionary new approaches in how to target specific genetic changes in the body and to try to change those problems at the source.”

– Dr. Adeline Vanderver, Program Director of Leukodystrophy Center of Excellence, Children’s Hospital of Philadelphia (CHOP)

“Our mission is to provide cutting-edge, clinical, multidisciplinary care to children living with leukodystrophy and to bring together researchers dedicated to solving these conditions and meaningfully advancing clinical care.”

Until a permanent cure is available, the hope is that the ongoing administration of the ASO treatment will stop further progression of the disease.