Neurological Disorders – Page 28

Molecular Characterization of hESC and hIPSC-Derived Spinal Motor Neurons

One of the main objectives of stem cell biology is to create physiologically relevant cell types that can be used to either facilitate the study of or directly treat human…

Phase 1/ 2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients with FOXG1 Syndrome (FS)

Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach

Stem Cell-Engineered Off-The-Shelf CAR-NKT Cell Therapy for Multiple Sclerosis

Late-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies

Microglia replacement therapy for CSF1R-related Leukoencephalopathy

CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

Development of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders

Dissecting cell-specific genetic and molecular drivers of amyotrophic lateral sclerosis for therapeutic insights