Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.
about
Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translationE Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal--Tailored Acceleration of AAV EvolutionLiver-targeted gene therapy: Approaches and challengesProgress and problems with the use of suicide genes for targeted cancer therapyPseudoxanthoma elasticumLysosomal storage disease: gene therapy on both sides of the blood-brain barrier.RNAe: an effective method for targeted protein translation enhancement by artificial non-coding RNA with SINEB2 repeat.Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVIIStrategies for targeting primate neural circuits with viral vectors.PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina.Recombinant adeno-associated virus vectors in the treatment of rare diseases.Obstacles and future of gene therapy for hemophilia.Gene editing technology as an approach to the treatment of liver diseases.Adeno-Associated Virus Gene Therapy for Liver Disease.Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I.Next-generation AAV vectors for clinical use: an ever-accelerating race.Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapyMoving forward toward a cure for hemophilia B.Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc-/- mice.Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy.Rational design for enhanced gene therapy with DNA transposonsHepatic lentiviral gene transfer prevents the long-term onset of hepatic tumours of glycogen storage disease type 1a in mice.Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.
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P2860
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.
description
2014 nî lūn-bûn
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2014年の論文
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2014年学术文章
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2014年学术文章
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2014年学术文章
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2014年学术文章
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2014年学术文章
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name
Liver-specific transcriptional ...... n mice and non-human primates.
@en
type
label
Liver-specific transcriptional ...... n mice and non-human primates.
@en
prefLabel
Liver-specific transcriptional ...... n mice and non-human primates.
@en
P2093
P2860
P50
P356
P1433
P1476
Liver-specific transcriptional ...... n mice and non-human primates.
@en
P2093
Abel Acosta-Sanchez
Amine Meliani
Caroline Le Guiner
Ermira Samara-Kuko
Federico Mingozzi
Ghislaine Cherel
Hanneke Evens
Inge Petrus
Janka Matrai
P2860
P304
P356
10.1038/MT.2014.114
P577
2014-06-23T00:00:00Z