Detalhe da pesquisa
1.
A modified mouse model of Friedreich's ataxia with conditional Fxn allele homozygosity delays onset of cardiomyopathy.
Am J Physiol Heart Circ Physiol
; 326(2): H357-H369, 2024 Feb 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38038720
2.
An Overview of Nonclinical and Clinical Liver Toxicity Associated With AAV Gene Therapy.
Toxicol Pathol
; 51(7-8): 400-404, 2023 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-37772805
3.
Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors.
Toxicol Pathol
; 50(1): 118-146, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34657529
4.
Zucker Lean Rats With Hepatic Steatosis Recapitulate Asymptomatic Metabolic Syndrome and Exhibit Greater Sensitivity to Drug-Induced Liver Injury Compared With Standard Nonclinical Sprague-Dawley Rat Model.
Toxicol Pathol
; 48(8): 994-1007, 2020 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33252024
5.
Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products.
Toxicol Pathol
; 46(8): 1020-1027, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30295175
6.
Nephron segment specific microRNA biomarkers of pre-clinical drug-induced renal toxicity: Opportunities and challenges.
Toxicol Appl Pharmacol
; 312: 34-41, 2016 Dec 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-26827821
7.
A road map to evaluate the proteome-wide selectivity of covalent kinase inhibitors.
Nat Chem Biol
; 10(9): 760-767, 2014 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-25038787
8.
Sequence motifs associated with hepatotoxicity of locked nucleic acid--modified antisense oligonucleotides.
Nucleic Acids Res
; 42(8): 4882-91, 2014 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-24550163
9.
Oral administration of soluble guanylate cyclase agonists to rats results in osteoclastic bone resorption and remodeling with new bone formation in the appendicular and axial skeleton.
Toxicol Pathol
; 43(3): 411-23, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25142129
10.
Identification of tubular injury microRNA biomarkers in urine: comparison of next-generation sequencing and qPCR-based profiling platforms.
BMC Genomics
; 15: 485, 2014 Jun 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-24942259
11.
Mechanistic investigations of test article-induced pancreatic toxicity at the endocrine-exocrine interface in the rat.
Toxicol Pathol
; 42(1): 229-42, 2014 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-24226507
12.
Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs.
Mol Ther Methods Clin Dev
; 29: 395-405, 2023 Jun 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37251978
13.
Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies.
Hum Gene Ther
; 33(21-22): 1126-1141, 2022 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35994386
14.
In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency.
Mol Ther Methods Clin Dev
; 24: 367-378, 2022 Mar 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35252470
15.
Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles.
Hum Gene Ther
; 33(3-4): 175-187, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34931542
16.
Outcomes of the European Federation of Pharmaceutical Industries and Associations Oligonucleotide Working Group Survey on Nonclinical Practices and Regulatory Expectations for Therapeutic Oligonucleotide Safety Assessment.
Nucleic Acid Ther
; 31(1): 7-20, 2021 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33054599
17.
Urinary miRNA Biomarkers of Drug-Induced Kidney Injury and Their Site Specificity Within the Nephron.
Toxicol Sci
; 180(1): 1-16, 2021 02 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-33367795
18.
Anti-IL5 decreases the number of eosinophils but not the severity of dermatitis in Sharpin-deficient mice.
Exp Dermatol
; 19(3): 252-8, 2010 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-19650867
19.
Acute lymphoid and gastrointestinal toxicity induced by selective p38alpha map kinase and map kinase-activated protein kinase-2 (MK2) inhibitors in the dog.
Toxicol Pathol
; 38(4): 606-18, 2010 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-20448081
20.
Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective.
Mol Ther Methods Clin Dev
; 19: 89-98, 2020 Dec 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33024793