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1 . Academic Journal
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
저자
by
Maria Dahl
;
Emma M.K. Smith
;
Sarah Warsi
;
Michael Rothe
;
Maria J. Ferraz
, et al.
소스
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 312-323 (2021)
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7 . Academic Journal
Looking into the Black Box: Insights into the Mechanisms of Somatic Cell Reprogramming
저자
by
Jeffrey L. Wrana
;
Azadeh Golipour
;
Laurent David
;
Payman Samavarchi-Tehrani
.
소스
Genes, Vol 2, Iss 1, Pp 81-106 (2011)
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6 .
Using IVIM/SAGA as screening tools during lentiviral vector lead selection for detection of clinically translatable insertional transformational risk
저자
by
Richard Pfeifer
;
Holly Horton
;
Azadeh Golipour
;
Essra Ridha
;
Axel Schambach
, et al.
소스
Molecular Genetics and Metabolism
. 138:107266
Web of Science
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7 .
The Guard1 clinical trial - A first in-human, phase 1/2 study evaluating AVR-RD-02, a hematopoietic stem cell (HSC) gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion
저자
by
Mathews Adera
;
Milena Veselinovic
;
Anita Treohan
;
Azadeh Golipour
;
Essra Ridha
.
소스
Molecular Genetics and Metabolism
. 138:106991
Web of Science
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7 .
High throughput monitoring of safety, potency and stability of gene therapy cell products in lysosomal disease patients
저자
by
Luca Biasco
;
Cristina Baricordi
;
Mariana Loperfido
;
Aimin Yan
;
Luigi Barbarossa
, et al.
소스
Molecular Genetics and Metabolism
. 135:S23
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7 .
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
저자
by
Christopher E. Mason
;
Daniella Pizzurro
;
Stefan Karlsson
;
Claudia Harper
;
Maria J. Ferraz
, et al.
소스
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 312-323 (2021)
Molecular Therapy-Methods and Clinical Development, 20, 312-323. CELL PRESS
Open Access (OpenAIRE)
Scopus
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7 .
Single-Cell Mapping of Human Hematopoietic Stem/Progenitor Cells States and Ex Vivo Dynamics: A Next Generation Approach to Stem Cell Gene Therapy Drug Product Characterization
저자
by
Luigi Barbarossa
;
Aimin Yan
;
Christopher E. Mason
;
Cristina Baricordi
;
Mariana Loperfido
, et al.
소스
Transplantation and Cellular Therapy
. 27:S34-S35
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7 .
The GuardOne clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease
저자
by
Josie Yang
;
Aneal Khan
;
Christopher E. Mason
;
Richard Pfeifer
;
Rachel Ciotti
, et al.
소스
Molecular Genetics and Metabolism
. 132:S51-S52
Web of Science
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7 .
A Comprehensive High Resolution Scrna-Seq Analytical Platform for Characterizing Human Hematopoietic Stem/Progenitor Cells States in Ex Vivo Drug Products
저자
by
Mercedes Segura
;
Cristina Baricordi
;
Christopher E. Mason
;
Azadeh Golipour
;
Luca Biasco
, et al.
소스
Blood
. 136:28-29
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7 .
Analysis of genetically engineered stem cell product and follow up of gene therapy patients through high-throughput single cell technologies
저자
by
Luca Biasco
;
Christopher E. Mason
;
Hareesh Chamarthi
;
Mariana Loperfido
;
Rachel Ciotti
, et al.
소스
Molecular Genetics and Metabolism
. 132:S18
Web of Science
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