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1 . Academic Journal
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia
저자
by
Bahal, Sameer
;
Zinicola, Marta
;
Moula, Shefta E
;
Whittaker, Thomas E.
;
Schejtman, Andrea
, et al.
소스
In
The Journal of Allergy and Clinical Immunology
July 2024 154(1):195-208
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7 . Academic Journal
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant
저자
by
Yeung, Jenny
;
Liao, Aiyin
;
Shaw, Matthew
;
Silva, Soraia
;
Vetharoy, Winston
, et al.
소스
In
Molecular Therapy
5 June 2024 32(6):1672-1686
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7 . Academic Journal
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV
저자
by
Vaidyanathan, Sriram
;
Kerschner, Jenny L.
;
Paranjapye, Alekh
;
Sinha, Vrishti
;
Lin, Brian
, et al.
소스
In
Molecular Therapy - Nucleic Acids
12 March 2024 35(1)
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7 . Academic Journal
An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells
저자
by
Rai, Rajeev
;
Naseem, Asma
;
Vetharoy, Winston
;
Steinberg, Zohar
;
Thrasher, Adrian J.
, et al.
소스
In
Molecular Therapy - Methods & Clinical Development
8 June 2023 29:58-69
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7 . Academic Journal
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy
저자
by
Sobrino, Steicy
;
Magnani, Alessandra
;
Semeraro, Michaela
;
Martignetti, Loredana
;
Cortal, Akira
, et al.
소스
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Cell Reports Medicine
21 February 2023 4(2)
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7 . Academic Journal
Alpharetroviral Vector–Mediated Gene Therapy for IL7RA-Deficient Severe Combined Immunodeficiency.
저자
by
Ha, Teng-Cheong
;
Morgan, Michael A.
;
Thrasher, Adrian J.
;
Schambach, Axel
.
소스
Human Gene Therapy
. 2024, Vol. 35 Issue 17/18, p669-679. 11p.
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6 . Academic Journal
Absence of γ-Chain in Keratinocytes Alters Chemokine Secretion, Resulting in Reduced Immune Cell Recruitment
저자
by
Nowak, Karolin
;
Linzner, Daniela
;
Thrasher, Adrian J.
;
Lambert, Paul F.
;
Di, Wei-Li
, et al.
소스
In
Journal of Investigative Dermatology
October 2017 137(10):2120-2130
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7 . Academic Journal
Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series
저자
by
Naviglio, Samuele
;
Cicalese, Maria Pia
;
Rivers, Elizabeth
;
Ferrua, Francesca
;
Bonfim, Carmem
, et al.
소스
In
Blood
July 2024
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7 . Academic Journal
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult
저자
by
Morris, Emma C.
;
Fox, Thomas
;
Chakraverty, Ronjon
;
Tendeiro, Rita
;
Snell, Katie
, et al.
소스
In
Blood
14 September 2017 130(11):1327-1335
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7 . Academic Journal
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells.
저자
by
Rai, Rajeev
;
Steinberg, Zohar
;
Romito, Marianna
;
Zinghirino, Federica
;
Hu, Yi-Ting
, et al.
소스
Human Gene Therapy
. Apr2024, Vol. 35 Issue 7/8, p269-283. 15p.
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