Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.

Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.

Item
http://www.wikidata.org/entity/Q42176546
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.Gene therapy for lysosomal storage disorders: a good start.Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC.Recent advances in gene therapy for lysosomal storage disorders.Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B miceHow close are we to therapies for Sanfilippo disease?Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.Targeting Root Cause by Systemic scAAV9-h Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice
P2860

Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.

Item
http://www.wikidata.org/entity/Q42176546
description
2014 nî lūn-bûn
@nan
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
2014年论文
@zh
2014年论文
@zh-cn
name
Biochemical, histological and ...... ebrospinal fluid gene therapy.
@en
Biochemical, histological and ...... ebrospinal fluid gene therapy.
@nl
type
label
Biochemical, histological and ...... ebrospinal fluid gene therapy.
@en
Biochemical, histological and ...... ebrospinal fluid gene therapy.
@nl
prefLabel
Biochemical, histological and ...... ebrospinal fluid gene therapy.
@en
Biochemical, histological and ...... ebrospinal fluid gene therapy.
@nl
P1433
P1476
P2093
P2860
P304
P31
P356
P433
P478
P50
P577
P5875
P698
P921
P356
P1433
P1476
Biochemical, histological and ...... ebrospinal fluid gene therapy.
@en
P2093
P2860
P304
P31
P356
P433
P478
P50
P577
P5875
P698
P921