000 02268ngm a2200457 a 4500
001 HST3807
003 UkLoHST
006 m c
007 cr|cna|||a||||
007 vz|czazum
008 141027s2014 enk|||||||||||s|||v|eng d
028 5 0 _a3807
_bHenry Stewart Talks
035 _a(UkLoHST)2892
040 _aUkLoHST
_beng
_cUkLoHST
100 1 _aBiffi, Alessandra,
_u(Telethon Institute for Gene Therapy, Italy)
_4spk
245 1 0 _aHematopoietic stem cell gene therapy for the treatment of metachromatic leukodystrophy
_h[electronic resource] /
_cAlessandra Biffi.
260 _aLondon :
_bHenry Stewart Talks,
_c2014.
300 _a1 online resource (1 streaming video file (37 min.) :
_bcolor, sound).
490 1 _aGene transfer and gene therapy,
_x2056-452X
500 _aAnimated audio-visual presentation with synchronized narration.
500 _aTitle from title frames.
505 0 _aContents: 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).
506 _aAccess restricted to subscribers.
538 _aMode of access: World Wide Web.
650 0 _aBrain
_xDiseases
_xGenetic aspects.
650 0 _aGene therapy.
650 0 _aMedical genetics.
650 0 _aStem cells
_xTherapeutic use.
650 2 _aGenetic Therapy.
650 2 _aGenetics, Medical.
650 2 _aHematopoietic Stem Cell Transplantation.
650 2 _aHematopoietic Stem Cells
_xmetabolism.
650 2 _aLentivirus.
650 2 _aLeukodystrophy, Metachromatic
_xgenetics.
650 2 _aLeukodystrophy, Metachromatic
_xtherapy.
650 2 _aMicroglia.
650 2 _aVirus Integration
_xgenetics.
830 0 _aHenry Stewart talks.
_pBiomedical & life sciences collection.
_pGene transfer and gene therapy.
856 4 0 _uhttps://hstalks.com/bs/2892/
856 4 2 _uhttps://hstalks.com/bs/p/808/
_3Series
999 _c77001
_d77001