912 results on '"Davidson, Beverly L."'
Search Results
2. Temporal restriction of Cas9 expression improves CRISPR-mediated deletion efficacy and fidelity
3. APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease
4. Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching
5. A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency
6. Correction for Morozko et al., PIAS1 modulates striatal transcription, DNA damage repair, and SUMOylation with relevance to Huntington’s disease
7. Antagonistic roles of canonical and Alternative-RPA in disease-associated tandem CAG repeat instability
8. PIAS1 modulates striatal transcription, DNA damage repair, and SUMOylation with relevance to Huntington’s disease
9. Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain
10. Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing
11. VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF
12. Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
13. DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencing
14. An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
15. Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain
16. Disease-Associated Short Tandem Repeats Co-localize with Chromatin Domain Boundaries
17. Cas9 nickase-mediated contraction of CAG/CTG repeats at multiple disease loci
18. AAV-based delivery of RNAi targeting Ataxin-2 improves survival, strength, and pathology in mouse models of rapidly and slowly progressive sporadic ALS
19. Regulated control of gene therapies by drug-induced splicing
20. Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary
21. Transcriptome sequencing reveals aberrant alternative splicing in Huntington's disease.
22. PIAS1 Regulates Mutant Huntingtin Accumulation and Huntington’s Disease-Associated Phenotypes In Vivo
23. A novel iPSC model reveals selective vulnerability of neurons in Multiple Sulfatase Deficiency
24. Reinstating Aberrant mTORC1 Activity in Huntington’s Disease Mice Improves Disease Phenotypes
25. APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease
26. Author Correction: Regulated control of gene therapies by drug-induced splicing
27. RNA Interference Improves Motor and Neuropathological Abnormalities in a Huntington's Disease Mouse Model
28. Allele-Specific Silencing of Dominant Disease Genes
29. Functional Correction of Established Central Nervous System Deficits in an Animal Model of Lysosomal Storage Disease with Feline Immunodeficiency Virus-Based Vectors
30. Unravelling Endogenous MicroRNA System Dysfunction as a New Pathophysiological Mechanism in Machado-Joseph Disease
31. CRISPR–Cas Gene Editing for Neurological Disease
32. Contributors
33. Recombinant Adeno-Associated Virus Type 2, 4, and 5 Vectors: Transduction of Variant Cell Types and Regions in the Mammalian Central Nervous System
34. Progressive Muscular Dystrophy in α-Sarcoglycan-Deficient Mice
35. Striatal neurons directly converted from Huntington’s disease patient fibroblasts recapitulate age-associated disease phenotypes
36. Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates
37. Dopaminergic Neurons Protected from Degeneration by GDNF Gene Therapy
38. Stimulation of New Bone Formation by Direct Transfer of Osteogenic Plasmid Genes
39. Standard screening methods underreport AAV-mediated transduction and gene editing
40. Publisher Correction: Lysosomal storage diseases
41. Lysosomal storage diseases
42. Author Correction: Lysosomal storage diseases
43. Antagonistic roles of canonical and alternative RPA in tandem CAG repeat diseases
44. Author Correction: Striatal neurons directly converted from Huntington’s disease patient fibroblasts recapitulate age-associated disease phenotypes
45. Huntington’s disease phenotypes are improved via mTORC1 modulation by small molecule therapy
46. Mapping PTBP splicing in human brain identifies targets for therapeutic splice switching including SYNGAP1
47. Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders
48. Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation
49. Doubling down on siRNAs in the brain
50. The Neuronal Ceroid Lipofuscinoses: Clinical Features and Molecular Basis of Disease
Catalog
Books, media, physical & digital resources
Discovery Service for Jio Institute Digital Library
For full access to our library's resources, please sign in.