761. Lentiviral Gene Therapy of Murine Hematopoietic Stem Cells Using a Codon Optimized Gene and Native IL2RG Promoter Region Corrects the SCID-X1 Phenotype

Molecular Therapy ◽

10.1016/s1525-0016(16)39119-5 ◽

2009 ◽

Vol 17 ◽

pp. S290

Keyword(s):

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Promoter Region ◽

Hematopoietic Stem ◽

Lentiviral Gene Therapy

Download Full-text

882. Lentiviral Gene Therapy of Hematopoietic Stem Cells Results in Phenotype Correction in a Mouse Model of Pompe Disease

Molecular Therapy ◽

10.1016/s1525-0016(16)40285-6 ◽

2008 ◽

Vol 16 ◽

pp. S329

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Hematopoietic Stem Cells ◽

Pompe Disease ◽

Hematopoietic Stem ◽

Lentiviral Gene Therapy

Download Full-text

530. Lentiviral Gene Therapy of Murine Hematopoietic Stem Cells Using Codon Optimized IL2RG cDNA: A Comparison of Multiple Promoter Elements and Transplant Conditions

Molecular Therapy ◽

10.1016/s1525-0016(16)37971-0 ◽

2010 ◽

Vol 18 ◽

pp. S204-S205

Keyword(s):

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Promoter Elements ◽

Hematopoietic Stem ◽

Lentiviral Gene Therapy ◽

Multiple Promoter

Download Full-text

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA

Case Medical Research ◽

10.31525/ct1-nct04201405 ◽

2019 ◽

Author(s):

Keyword(s):

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Mucopolysaccharidosis Type ◽

Hematopoietic Stem ◽

Type Iiia ◽

Mucopolysaccharidosis Type Iiia

Download Full-text

Development of gene therapy for hematopoietic stem cells

Scandinavian Journal of Clinical and Laboratory Investigation ◽

10.1080/003655102320754700 ◽

2002 ◽

Vol 62 (5) ◽

pp. 10-10

Keyword(s):

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Hematopoietic Stem

Download Full-text

Faculty Opinions recommendation of Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.739368425.793582178 ◽

2021 ◽

Author(s):

Frank Staal

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Hematopoietic Stem Cells ◽

Lentiviral Vector ◽

Hematopoietic Stem ◽

Diamond Blackfan Anemia ◽

Download Full-text

Human Hematopoietic Stem Cells, Progenitors, and Peripheral Blood Lymphocytes as Targets for the Correction of Immune System Disorders via Gene Therapy

Molecular Biology of B-Cell and T-Cell Development ◽

10.1007/978-1-4757-2778-4_27 ◽

1998 ◽

pp. 545-571

Author(s):

Karen E. Pollok ◽

David A. Williams

Keyword(s):

Gene Therapy ◽

Immune System ◽

Hematopoietic Stem Cells ◽

Peripheral Blood ◽

Peripheral Blood Lymphocytes ◽

Hematopoietic Stem ◽

Blood Lymphocytes

Download Full-text

Gene Therapy Using Hematopoietic Stem Cells

Hematopoietic Stem Cell Transplantation ◽

10.1201/b14000-34 ◽

2000 ◽

pp. 542-557

Keyword(s):

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Hematopoietic Stem

Download Full-text

Experimental Approaches to Hemophilia Gene Therapy: Gene Transfer into Hematopoietic Stem Cells

33rd Hemophilia Symposium ◽

10.1007/978-3-642-18260-0_21 ◽

2004 ◽

pp. 153-158

Author(s):

A. Tiede ◽

M. Eder ◽

M. Scherr ◽

A. Ganser ◽

M. von Depka Prondzinski

Keyword(s):

Gene Therapy ◽

Gene Transfer ◽

Hematopoietic Stem Cells ◽

Hematopoietic Stem ◽

Experimental Approaches

Download Full-text

Genetically Modified Hematopoietic Stem Cells for Gene Therapy in Beta-Thalassemia: Power of Viral Vector Towards Clinical Application

10.2174/9789815036756121010011 ◽

2021 ◽

pp. 133-144

Keyword(s):

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Clinical Application ◽

Viral Vector ◽

Genetically Modified ◽

Beta Thalassemia ◽

Hematopoietic Stem

Download Full-text

407. A Clinically-Relevant Gene Therapy Strategy for Hemophilia A: Non-Myeloablative Transplantation of Hematopoietic Stem Cells Transduced with a High-Expression Factor VIII Transgene

Molecular Therapy ◽

10.1016/s1525-0016(16)44613-7 ◽

2007 ◽

Vol 15 ◽

pp. S157

Keyword(s):

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Factor Viii ◽

Hemophilia A ◽

High Expression ◽

Hematopoietic Stem ◽

Therapy Strategy ◽

Gene Therapy Strategy ◽

Download Full-text