Stephanie Cherqui, PhD, on Bringing Attention to Cystinosis and Gene Therapy
The associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.
Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD
The Potential Impact of Gene and Cell Therapy in Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.