Detalhe da pesquisa
1.
Antagonistic roles of canonical and Alternative-RPA in disease-associated tandem CAG repeat instability.
Cell
; 186(22): 4898-4919.e25, 2023 10 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-37827155
2.
Disease-Associated Short Tandem Repeats Co-localize with Chromatin Domain Boundaries.
Cell
; 175(1): 224-238.e15, 2018 09 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-30173918
3.
Singles engage the RNA interference pathway.
Cell
; 150(5): 873-5, 2012 Aug 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-22939614
4.
Regulated control of gene therapies by drug-induced splicing.
Nature
; 596(7871): 291-295, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34321659
5.
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease.
Mol Ther
; 32(5): 1373-1386, 2024 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38504517
6.
A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency.
Mol Genet Metab
; 141(2): 108116, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-38161139
7.
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain.
Mol Ther
; 31(3): 760-773, 2023 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36617193
8.
PIAS1 modulates striatal transcription, DNA damage repair, and SUMOylation with relevance to Huntington's disease.
Proc Natl Acad Sci U S A
; 118(4)2021 01 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-33468657
9.
Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics.
Hum Mol Genet
; 30(24): 2469-2487, 2021 11 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-34296279
10.
Regional Variation of Splicing QTLs in Human Brain.
Am J Hum Genet
; 107(2): 196-210, 2020 08 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-32589925
11.
Author Correction: Regulated control of gene therapies by drug-induced splicing.
Nature
; 602(7897): E25, 2022 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-35105975
12.
Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation.
Mol Ther
; 30(7): 2464-2473, 2022 07 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35395398
13.
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.
Mol Ther
; 30(8): 2646-2663, 2022 08 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-35690906
14.
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Mol Ther
; 30(7): 2416-2428, 2022 07 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35585789
15.
Gene therapy for ALS: A review.
Mol Ther
; 29(12): 3345-3358, 2021 12 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33839324
16.
Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary.
Mol Ther
; 29(12): 3332-3344, 2021 12 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34547463
17.
The long non-coding RNA NEAT1 is elevated in polyglutamine repeat expansion diseases and protects from disease gene-dependent toxicities.
Hum Mol Genet
; 27(24): 4303-4314, 2018 12 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30239724
18.
AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity.
Mol Ther
; 27(2): 465-478, 2019 02 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30559071
19.
Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy.
Mol Ther
; 26(4): 1118-1126, 2018 04 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-29503202
20.
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates.
Mol Ther
; 31(3): 609-612, 2023 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36610400