Adrian J Thrasher
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Explore the profile of Adrian J Thrasher including associated specialties, affiliations and a list of published articles.
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333
Citations
14515
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Recent Articles
11.
Vaidyanathan S, Kerschner J, Paranjapye A, Sinha V, Lin B, Bedrosian T, et al.
Mol Ther Nucleic Acids
. 2024 Feb;
35(1):102134.
PMID: 38384445
A "universal strategy" replacing the full-length cDNA may treat >99% of people with cystic fibrosis (pwCF), regardless of their specific mutations. Cas9-based gene editing was used to insert the cDNA...
12.
Rai R, Steinberg Z, Romito M, Zinghirino F, Hu Y, White N, et al.
Hum Gene Ther
. 2024 Jan;
35(7-8):269-283.
PMID: 38251667
Interleukin 7 Receptor alpha Severe Combined Immunodeficiency (IL7R-SCID) is a life-threatening disorder caused by homozygous mutations in the gene. Defective IL7R expression in humans hampers T cell precursors' proliferation and...
13.
Westhaus A, Eamegdool S, Fernando M, Fuller-Carter P, Brunet A, Miller A, et al.
Sci Rep
. 2023 Dec;
13(1):21946.
PMID: 38081924
Adeno-associated viral (AAV) vector-mediated retinal gene therapy is an active field of both pre-clinical as well as clinical research. As with other gene therapy clinical targets, novel bioengineered AAV variants...
14.
Whittaker T, Moula S, Bahal S, Bakri F, Hayajneh W, Daoud A, et al.
Hum Gene Ther
. 2023 Dec;
35(7-8):298-312.
PMID: 38062734
Replacing a faulty gene with a correct copy has become a viable therapeutic option as a result of recent progress in gene editing protocols. Targeted integration of therapeutic genes in...
15.
Georgiadis A, Tschernutter M, Bainbridge J, Balaggan K, Mowat F, West E, et al.
PLoS One
. 2023 Dec;
18(12):e0295782.
PMID: 38060570
[This corrects the article DOI: 10.1371/journal.pone.0015730.].
16.
Rai R, Naseem A, Vetharoy W, Steinberg Z, Thrasher A, Santilli G, et al.
Mol Ther Methods Clin Dev
. 2023 Mar;
29:58-69.
PMID: 36950452
Gene editing has emerged as a powerful tool for the therapeutic correction of monogenic diseases. CRISPR-Cas9 applied to hematopoietic stem and progenitor cells (HSPCs) has shown great promise in proof-of-principle...
17.
Westhaus A, Cabanes-Creus M, Dilworth K, Zhu E, Salas Gomez D, Navarro R, et al.
Hum Gene Ther
. 2023 Mar;
34(7-8):273-288.
PMID: 36927149
The liver is a prime target for gene therapies using recombinant adeno-associated viral vectors. Multiple clinical trials have been undertaken for this target in the past 15 years; however, we...
18.
Sobrino S, Magnani A, Semeraro M, Martignetti L, Cortal A, Denis A, et al.
Cell Rep Med
. 2023 Jan;
4(2):100919.
PMID: 36706754
X-linked chronic granulomatous disease (CGD) is associated with defective phagocytosis, life-threatening infections, and inflammatory complications. We performed a clinical trial of lentivirus-based gene therapy in four patients (NCT02757911). Two patients...
19.
Topal J, Panchal N, Barroeta A, Roppelt A, Mudde A, Gaspar H, et al.
J Clin Immunol
. 2022 Nov;
43(2):440-451.
PMID: 36329240
Background: X-linked inhibitor of apoptosis protein (XIAP) deficiency is a severe immunodeficiency with clinical features including hemophagocytic lymphohistiocytosis (HLH) and inflammatory bowel disease (IBD) due to defective NOD2 responses. Management...
20.
Garcia M, Bonafont J, Martinez-Palacios J, Xu R, Turchiano G, Svensson S, et al.
Mol Ther Methods Clin Dev
. 2022 Oct;
27:96-108.
PMID: 36212909
Recessive dystrophic epidermolysis bullosa, a devastating skin fragility disease characterized by recurrent skin blistering, scarring, and a high risk of developing squamous cell carcinoma is caused by mutations in ,...