Preclinical efficacy and safety evaluation of BD211 autologous CD34+ hematopoietic stem cell injection for transfusion-dependent β-thalassemia in NCG-X mice

Customizable virus-like particles deliver CRISPR-Cas9 ribonucleoprotein for effective ocular neovascular and Hungtington's disease gene therapy

Identification of restrictive molecules involved in oncolytic virotherapy using genome-wide CRISPR screening

In vivo CRISPR gene editing in patients with herpetic stromal keratitis.

Modified lentiviral globin gene therapy for pediatric β0/β0 transfusion-dependent β-thalassemia: A single-center, single-arm pilot trial

Dendritic-cell-targeting virus-like particles as potent mRNA vaccine carriers