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Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
Florian Eichler
, Christine Duncan
, Patricia L. Musolino
,
Paul J. Orchard
, Satiro De Oliveira
, Adrian J. Thrasher
, Myriam Armant
, Colleen Dansereau
,
Troy C. Lund
, Weston P. Miller
, Gerald V. Raymond
, Raman Sankar
, Ami J. Shah
, Caroline Sevin
, H. Bobby Gaspar
, Paul Gissen
, Hernan Amartino
, Drago Bratkovic
, Nicholas J.C. Smith
, Asif M. Paker
Esther Shamir, Tara O’Meara, David Davidson, Patrick Aubourg, David A. Williams
Show 5 others
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Pediatric Blood & Marrow Transplant & Cellular Therapy
Neurology
Center for Genome Engineering
Transplant and Cellular Therapy
Research output
:
Contribution to journal
›
Article
›
peer-review
513
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Scopus citations
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Keyphrases
Adrenoleukodystrophy
100%
Hematopoietic Stem Cell Gene Therapy
100%
Disease Progression
75%
Magnetic Resonance Imaging
75%
Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT)
75%
Lentis
75%
Functional Disability
75%
Graft-versus-host Disease (GvHD)
50%
Gene Therapy
50%
Neurological Function
50%
Interim Analysis
50%
Efficacy Study
25%
Safety Study
25%
Lentiviral Vector
25%
Long-term Safety
25%
Neurodegeneration
25%
Loss Function
25%
Effective Alternative
25%
Engraftment
25%
Oncogene
25%
Duration of Response
25%
Demyelination
25%
Clinical Symptoms
25%
CD34+ Cells
25%
X-linked Adrenoleukodystrophy (X-ALD)
25%
Early Disease
25%
Single Group
25%
Late Gadolinium Enhancement
25%
Safer Alternatives
25%
Functional Change
25%
Transplant Complications
25%
Treatment-related Mortality
25%
Neurological Deterioration
25%
ABCD1
25%
Investigational Therapies
25%
Neurologic Death
25%
Disease Enhancement
25%
Medicine and Dentistry
Hematopoietic Cell
100%
Adrenoleukodystrophy
100%
Stem Cell Gene Therapy
100%
Magnetic Resonance Imaging
60%
Disease Exacerbation
60%
Gene Therapy
40%
Allogeneic Stem Cell Transplantation
40%
Infusion
40%
Interim Analysis
40%
Graft Versus Host Reaction
40%
Engraftment
20%
Efficacy Study
20%
Deterioration
20%
Neurodegeneration
20%
Lentivirus Vector
20%
Demyelination
20%
Oncogene
20%
Allogeneic Hematopoietic Stem Cell Transplantation
20%
Experimental Therapy
20%
ABCD1
20%
Elivaldogene Tavalentivec
20%
Gadolinium
20%
Diseases
20%
Clinical Symptom
20%
Biochemistry, Genetics and Molecular Biology
Gene Therapy
100%
Hematopoietic Cell
100%
Adrenoleukodystrophy
100%
Magnetic Resonance Imaging
60%
Allogeneic Stem Cell Transplantation
40%
Oncogene
20%
Efficacy Study
20%
Gadolinium
20%
Lentivirus Vector
20%
CD34
20%
Allogeneic Hematopoietic Stem Cell Transplantation
20%
ABCD1
20%
Elivaldogene Tavalentivec
20%