Detalhe da pesquisa
1.
Lentiviral Gene Therapy for Artemis-Deficient SCID.
N Engl J Med;
387(25): 2344-2355, 2022 12 22.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36546626
2.
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine.
Mol Ther;
31(4): 1123-1135, 2023 04 05.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36710491
3.
Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II.
Mol Genet Metab;
138(4): 107539, 2023 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37023503
4.
Contribution of the innate and adaptive immune systems to aortic dilation in murine mucopolysaccharidosis type I.
Mol Genet Metab;
135(3): 193-205, 2022 03.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35165009
5.
Correction of Fanconi Anemia Mutations Using Digital Genome Engineering.
Int J Mol Sci;
23(15)2022 Jul 29.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35955545
6.
Biodistribution of Adeno-Associated Virus Serotype 5 Viral Vectors Following Intrathecal Injection.
Mol Pharm;
18(10): 3741-3749, 2021 10 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34460254
7.
Differences in MPS I and MPS II Disease Manifestations.
Int J Mol Sci;
22(15)2021 Jul 23.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34360653
8.
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
Mol Ther;
27(1): 178-187, 2019 01 02.
Artigo
em Inglês
| MEDLINE
| ID: mdl-30528089
9.
Dysostosis Multiplex in Human Mucopolysaccharidosis Type 1 H and in Animal Models of the Disease.
Pediatr Endocrinol Rev;
17(4): 317-326, 2020 Aug.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32780955
10.
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
Mol Ther;
26(4): 1127-1136, 2018 04 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29580682
11.
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice.
Mol Ther Methods Clin Dev;
32(1): 101201, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38374962
12.
The Sleeping Beauty transposon system: a non-viral vector for gene therapy.
Hum Mol Genet;
20(R1): R14-20, 2011 Apr 15.
Artigo
em Inglês
| MEDLINE
| ID: mdl-21459777
13.
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction.
Hum Gene Ther;
34(1-2): 8-18, 2023 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36541357
14.
Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome.
Mol Genet Metab Rep;
34: 100956, 2023 Mar.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36704405
15.
AAV-mediated gene transfer to colon-innervating primary afferent neurons.
Front Pain Res (Lausanne);
4: 1225246, 2023.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37599864
16.
Adeno-associated virus-mediated gene transfer of arginine decarboxylase to the central nervous system prevents opioid analgesic tolerance.
Front Pain Res (Lausanne);
4: 1269017, 2023.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38405182
17.
Cas9-induced targeted integration of large DNA payloads in primary human T cells via homology-mediated end-joining DNA repair.
Nat Biomed Eng;
2023 Dec 13.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38092857
18.
Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration.
Mol Genet Metab;
106(1): 131-4, 2012 May.
Artigo
em Inglês
| MEDLINE
| ID: mdl-22420937
19.
Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy.
Expert Opin Biol Ther;
22(9): 1151-1162, 2022 09.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36107226
20.
Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells.
Hum Gene Ther;
33(23-24): 1279-1292, 2022 12.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36226412