Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction
about
Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemiaImpact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transductionPre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemiaGenetic disorders of vitamin B12 metabolism: eight complementation groups – eight genesNon-cell-autonomous activation of IL-6/STAT3 signaling mediates FGF19-driven hepatocarcinogenesis.Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia.Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.Perinatal gene transfer to the liver.AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene deliveryAAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in miceNeutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.Vitamin B(12) metabolism during pregnancy and in embryonic mouse models.Adeno-associated virus-mediated rescue of neonatal lethality in argininosuccinate synthetase-deficient miceAdvances and challenges in the treatment of branched-chain amino/keto acid metabolic defects.Improving lipoprotein profiles by liver-directed gene transfer of low density lipoprotein receptor gene in hypercholesterolaemia mice.Altering α-dystroglycan receptor affinity of LCMV pseudotyped lentivirus yields unique cell and tissue tropismRepeatable, Inducible Micro-RNA-Based Technology Tightly Controls Liver Transgene Expression.
P2860
Q26865440-BEE3BFA5-9ABF-4660-9998-B8E473B813B6Q27307010-BDD64B5C-7021-4A48-BEB6-19FB38564340Q28276617-EBB43C93-9690-4D04-A3BB-CDDB5B642C62Q28295609-8190E52A-F938-44A8-AB33-61E475F6013CQ29541652-A731E6B6-FEFA-4A19-9C71-483F6A2D4D3BQ33716459-5E0EB632-52A1-41DB-82D5-176EB6B16FEBQ34774811-ACA67566-2D5A-4E85-8FDB-49093EB0EEC3Q35056714-025326DF-B3B0-4767-9279-55D00B42B63DQ35238390-B9A5844A-88CD-4684-B934-CFEE1B272E9CQ35568520-4E3F14AA-C923-433B-9C44-AD487F8E2417Q35988663-D37C124E-D69E-43A5-89F5-7228608F6ADAQ36055431-FB2ED1F4-B865-4E00-BB01-BD5D360943A6Q36203308-7BE97220-8E45-40AD-ADCE-416DBE55C599Q36825929-CD7E8FC3-7B75-43BB-ACFC-6811F7B857E8Q37235374-6FEAEDC2-303B-4076-B8A6-252C8D3C4F23Q37254867-6C223D81-3916-4C3F-BA75-4C500BBF8EE8Q37835047-A9CC4C52-2273-4F72-97C3-88F36989FD60Q40678976-7320A54F-1952-40A8-BA84-C4D4940D1623Q41980779-94A09956-065D-4208-95B3-60BC9699F473Q42768559-D822082A-5533-438F-B0F2-16BB51F3B511
P2860
Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction
description
2010 nî lūn-bûn
@nan
2010 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած
@hyw
2010 թվականի սեպտեմբերին հրատարակված գիտական հոդված
@hy
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
name
Liver-directed recombinant ade ...... ong-term phenotypic correction
@ast
Liver-directed recombinant ade ...... ong-term phenotypic correction
@en
type
label
Liver-directed recombinant ade ...... ong-term phenotypic correction
@ast
Liver-directed recombinant ade ...... ong-term phenotypic correction
@en
prefLabel
Liver-directed recombinant ade ...... ong-term phenotypic correction
@ast
Liver-directed recombinant ade ...... ong-term phenotypic correction
@en
P2860
P356
P1433
P1476
Liver-directed recombinant ade ...... ong-term phenotypic correction
@en
P2093
Charles P Venditti
Suma Chandrasekaran
P2860
P304
P356
10.1089/HUM.2010.008
P577
2010-09-01T00:00:00Z