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2. Gene therapy approaches for familial ALS

3. AAV9-mediated SMN expression restricted to theCNS does not rescue SMA mice

5. Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes

6. A new AAV10-mediated gene therapy for SOD1 -linked ALS

8. Implication of the SMN complex in the biogenesis and steady state level of the Signal Recognition Particle

9. rAAV9-mediated gene transfer in the spinal cord of a feline model of motor neuron degeneration

11. AAV1-, AAV2- and AAV5-Mediated human alpha-Iduronidase Gene Transfer In The Brain of Nonhuman Primate: Vector Diffusion and Bio Distribution

18. G.O.20

30. Neuronal transfer of the human Cu/Zn superoxide dismutase gene increases the resistance of dopaminergic neurons to 6-hydroxydopamine.

31. Adenovirus for neurodegenerative diseases: in vivo strategies and ex vivo gene therapy using human neural progenitors

34. Splicing efficiency of minor introns in a mouse model of SMA predominantly depends on their branchpoint sequence and can involve the contribution of major spliceosome components.

35. Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.

36. AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.

37. [SMA: from gene discovery to gene therapy].

38. Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models.

39. Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice.

40. β-Amyloid Precursor Protein Intracellular Domain Controls Mitochondrial Function by Modulating Phosphatase and Tensin Homolog-Induced Kinase 1 Transcription in Cells and in Alzheimer Mice Models.

41. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

42. A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.

43. SECIS-binding protein 2 interacts with the SMN complex and the methylosome for selenoprotein mRNP assembly and translation.

44. A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome.

45. Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

46. Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology.

47. Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins.

48. Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases.

49. Moving towards treatments for spinal muscular atrophy: hopes and limits.

50. Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice.

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