Generation of HEXA-deficient hiPSCs from fibroblasts of a Tay

Human iPSC line TSD-01-hiPSC was generated from fibroblasts of a patient with infantile Tay-Sachs disease (TSD). The patient is compound heterozygous …

Rui Zhao's research works University of Alabama at Birmingham, AL (UAB) and other places

Frontiers Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies

Modeling neuronopathic storage diseases with patient-derived culture systems. - Abstract - Europe PMC

Presynaptic Dysfunction in Neurons Derived from Tay–Sachs iPSCs - ScienceDirect

Engineering of Human-Induced Pluripotent Stem Cells for Precise Disease Modeling

Frontiers Human Motor Neurons With SOD1-G93A Mutation Generated From CRISPR/Cas9 Gene-Edited iPSCs Develop Pathological Features of Amyotrophic Lateral Sclerosis

Presynaptic Dysfunction in Neurons Derived from Tay–Sachs iPSCs - ScienceDirect

Generation of HEXA-deficient hiPSCs from fibroblasts of a Tay-Sachs disease patient - ScienceDirect

Intranasal Delivery of CRISPR-Cas9 Edited HEXA Gene to Treat Tay Sachs Disease in Newborn HEXA/NEU3-Deficient Mice — Le Pouls

Presynaptic Dysfunction in Neurons Derived from Tay–Sachs iPSCs - ScienceDirect