Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies
Neuronopathic Gaucher disease (nGD) is a lysosomal storage disorder caused by GBA1 mutations, leading to defective acid β-glucosidase (GCase) and accumulation of glycosphingolipid substrates, causing…
Read the full articleYou might also wanna read
Single-cell multi-omic atlas and morphogen screening informs midbrain and hindbrain organoid engineering
A Nature study reveals 1-cell transcriptome & chromatin sequence mapping to engineer mid/hindbrain organoids, aiding neuro disorder study. P
Advances in Brain-on-a-Chip and BBB-on-a-Chip Models for Neurodegenerative Disease Research
Brain-on-a-chip is a microphysiologic platform that comprises cultured brain cells to understand brain disease pathogenesis and treatment. T
journals.sagepub.com·15d agoA Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Injection of LY-N001 Injection for the Treatment of Moderate to Advanced Parkinson's Disease With GBA1 Mutations
Conditions : Parkinson's Disease (PD) Interventions : Genetic: LY-N001 Injection Sponsors : Lingyi Biotech Co., Ltd. Not yet recruiting
Hematopoietic stem cell gene therapy delivers cell-penetrating frataxin to treat Friedreich's ataxia in preclinical models
Engineered hematopoietic stem cells delivering a secreted, tissue-penetrating frataxin restore mitochondrial function and ameliorate disease
Emerging therapeutic strategies for mitochondrial DNA-related primary mitochondrial diseases: a review of gene editing, heteroplasmy modulation, and transplantation approaches
In this review, Shi et al. discuss emerging therapeutic strategies for mitochondrial DNA-related primary mitochondrial diseases, including g
Nutrigenomics framework identifies vitamin B3 as potential therapy for NAXD deficiency disorder
A nutrigenomics framework identifies genetic diseases amenable to vitamin B2 and B3 therapies. This approach nominates vitamin B3 as a thera
Comments
Sign in to join the conversation.
No comments yet. Be the first.