Search Results - "stem cell"

Hematopoietic stem cell gene therapy for the treatment of metachromatic leukodystrophy

Table of Contents: “…Contents: Metachromatic Leukodystrophy (MLD) -- Hematopoietic stem cell therapy (HCT) for lysosomal storage disorders (LSDs) -- Improving HCT efficacy -- The role of brain pre-conditioning -- Mechanisms of microglia turnover -- Gene therapy (GT) & enzyme release -- Gene therapy in murine models -- HSC gene therapy for MLD -- Lentivirus integration site analysis -- Stem cells analysis -- ARSA activity and delivery to the CNS -- Cerebral and peripheral demyelination -- Motor function testing (GMFM).…”
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The ethics and regulation of cell and tissue therapies in the UK

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ATMPs from academia to industry getting it right first time (GIRFT) /

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Surface-mediated targeting of lentiviral vectors

Table of Contents: “…Contents: In vivo and ex vivo gene therapy -- Cell entry modes of retroviral vectors -- Molecular functions of viral envelope proteins -- Introduction to surface targeting -- Targeting subtypes of lymphocytes and hematopoietic stem cells (Ex vivo) -- In vivo targeting (local vs. systemic administration).…”
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Lentiviral vectors design, biological properties, milestones and current major applications, hazards. Part 2 of 2 /

Table of Contents: “…BMT -- HSC gene transfer by LV: current issues -- Restriction factors for lentiviral gene transfer -- Ex vivo transduction by lentiviral vectors -- HSC gene transfer by LV: current goals -- Applications: ex vivo into hematopoietic stem cells, ex vivo into lymphocytes, In vivo gene transfer (liver and CNS) -- Systemic delivery of LV: current issues -- Direct LV injection into the CNS -- CNS gene transfer: current issues.…”
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Development and regulation of cellular and gene therapy products FDA perspective /

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CRISPR editing therapy for Duchenne Muscular Dystrophy part 1 of 2 /

Table of Contents: “…Contents: Duchene Muscular Dystrophy (DMD) -- Dystrophin function -- MDX mouse model -- AAV CRISPR local therapy in mice -- AAV CRISPR systemic therapy in mice -- Myogenic stem cells can be transduced and edited by AAV9 CRISPR therapy.…”
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Helper-dependent adenoviral vectors for gene therapy

Table of Contents: “…Contents: Overview of gene therapy vectors -- Helper-Dependent Ad (HDAd) vector -- Liver and lung directed gene therapy -- Hemophilia B gene therapy -- OTC deficiency clinical trial -- Balloon occlusion catheter-based delivery of HDAd -- HDAd-mediated CF gene therapy -- Brain gene therapy -- HDAd as genetic vaccine -- HDAd and stem cells.…”
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Mechanistic aspects and therapeutic applications of RNAi

Table of Contents: “…Contents: RNAi mechanisms -- RNAi and HIV infection -- Lentiviral vector-mediated transduction of stem cells and T-cells -- Gene therapy based treatment of HIV-1 -- HIV mutants that abrogate RNAi -- Multiplexing different siRNAs and siRNAs with other antiviral RNAs to minimize viral resistance -- An HIV Tat inducible shRNA expression system -- Multiplexing shRNA with dominant negative M10 protein inhibition of HIV.…”
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