RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy.
about
Noninvasive and targeted gene delivery into the brain using microbubble-facilitated focused ultrasound.Strategies to rescue the consequences of inducible arginase-1 deficiency in miceStructure of neurotropic adeno-associated virus AAVrh.8.Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies.Experimental therapies in the murine model of globoid cell leukodystrophy.Minimal ureagenesis is necessary for survival in the murine model of hyperargininemia treated by AAV-based gene therapy.Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophyAAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient mice.ATP7A gene addition to the choroid plexus results in long-term rescue of the lethal copper transport defect in a Menkes disease mouse modelEfficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouseSystemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal miceAugmentation of transgene-encoded protein after neonatal injection of adeno-associated virus improves hepatic copy number without immune responses.Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery.AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in miceIn utero copper treatment for Menkes disease associated with a severe ATP7A mutationLong-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher MiceAAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse.Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy.Lethal phenotype in conditional late-onset arginase 1 deficiency in the mouse.Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplantLong-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery.Adeno-Associated Viral Vector Delivery to the Enteric Nervous System: A Review.Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis.Optimized AAV rh.10 Vectors That Partially Evade Neutralizing Antibodies during Hepatic Gene Transfer.Spinal nociceptive circuit analysis with recombinant adeno-associated viruses: the impact of serotypes and promoters.The influence of rAAV2-mediated SOX2 delivery into neonatal and adult human RPE cells; a comparative study.Transduction efficiency of neurons and glial cells by AAV-1, -5, -9, -rh10 and -hu11 serotypes in rat spinal cord following contusion injury.Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector.Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy.AAV-encoded OTC activity persisting to adulthood following delivery to newborn spf(ash) mice is insufficient to prevent shRNA-induced hyperammonaemia.Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats.Adeno-associated virus serotype rh10 is a useful gene transfer vector for sensory nerves that innervate bone in immunodeficient mice.
P2860
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P2860
RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy.
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
RH10 provides superior transge ...... pes for neonatal gene therapy.
@ast
RH10 provides superior transge ...... pes for neonatal gene therapy.
@en
RH10 provides superior transge ...... pes for neonatal gene therapy.
@nl
type
label
RH10 provides superior transge ...... pes for neonatal gene therapy.
@ast
RH10 provides superior transge ...... pes for neonatal gene therapy.
@en
RH10 provides superior transge ...... pes for neonatal gene therapy.
@nl
prefLabel
RH10 provides superior transge ...... pes for neonatal gene therapy.
@ast
RH10 provides superior transge ...... pes for neonatal gene therapy.
@en
RH10 provides superior transge ...... pes for neonatal gene therapy.
@nl
P2860
P356
P1476
RH10 provides superior transge ...... pes for neonatal gene therapy.
@en
P2093
Chuhong Hu
Gerald S Lipshutz
P2860
P304
P356
10.1002/JGM.1496
P577
2010-09-01T00:00:00Z