Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
A Biffi, E Montini, L Lorioli, M Cesani, F Fumagalli… - Science, 2013 - science.org
Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
A Biffi, E Montini, L Lorioli, M Cesani, F Fumagalli… - Science, 2013 - experts.nebraska.edu
Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by
arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive motor and …
arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive motor and …
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
A Biffi, E Montini, L Lorioli, M Cesani, F Fumagalli… - Science, 2013 - cir.nii.ac.jp
抄録< jats: title> Next-Generation Gene Therapy</jats: title>< jats: p> Few disciplines in
contemporary clinical research have experienced the high expectations directed at the gene …
contemporary clinical research have experienced the high expectations directed at the gene …
[PDF][PDF] Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits
M Leukodystrophy - Science, 2013 - openaccessrepository.it
Methods: We optimized LV manufacturing and HSC transduction in clinical grade conditions.
Three children with ARSA deficiency and mutations associated with early-onset MLD were …
Three children with ARSA deficiency and mutations associated with early-onset MLD were …
[PDF][PDF] Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits
M Leukodystrophy - Science, 2013 - researchgate.net
Methods: We optimized LV manufacturing and HSC transduction in clinical grade conditions.
Three children with ARSA deficiency and mutations associated with early-onset MLD were …
Three children with ARSA deficiency and mutations associated with early-onset MLD were …
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
A Biffi, E Montini, L Lorioli, M Cesani, F Fumagalli… - SCIENCE, 2013 - research.unipg.it
Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by
arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive motor and …
arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive motor and …
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
A Biffi, E Montini, L Lorioli, M Cesani… - … (New York, NY), 2013 - pubmed.ncbi.nlm.nih.gov
Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by
arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive motor and …
arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive motor and …
[PDF][PDF] Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits
M Leukodystrophy - Science, 2013 - academia.edu
Methods: We optimized LV manufacturing and HSC transduction in clinical grade conditions.
Three children with ARSA deficiency and mutations associated with early-onset MLD were …
Three children with ARSA deficiency and mutations associated with early-onset MLD were …
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
A Biffi, E Montini, L Lorioli, M Cesani… - Science (New York …, 2013 - europepmc.org
A comment on this article appears in" Broadening the indications for hematopoietic stem cell
genetic therapies." Cell Stem Cell. 2013 Sep 5; 13 (3): 263-4. A comment on this article …
genetic therapies." Cell Stem Cell. 2013 Sep 5; 13 (3): 263-4. A comment on this article …