Results 121 to 130 of about 1,008,618 (142)

The best of both worlds: AAV-mediated gene transfer empowered by LNP delivery of Sleeping Beauty transposase for durable transgene expression in vivo. [PDF]

Mol Ther
Sandoval-Villegas N, Ivics Z.
europepmc   +1 more source

Preclinical pharmacology and toxicology study of an AAV8-tATP7B vector for Wilson's disease. [PDF]

Clin Mol Hepatol
Wang Z, Wang M, Wu M, Ye L, Lou T, Sun X, Zhao W, Liu F, Ren T, Li W, Fan JG.   +10 more
europepmc   +1 more source

Nipah Virus Encephalitis: Pathogenetic Aspects and Current Therapeutic Strategies. [PDF]

Pathogens
Scotto G, Fazio V, Moula AM, Bavisetty SCB, Franza A, Massa S.   +5 more
europepmc   +1 more source

Advancements in CRISPR-based <i>in vivo</i> gene therapy for hemophilia. [PDF]

Front Genome Ed
Zhang X, Xu K, Zou S, Zhu Z, Teng S, Huang P.   +5 more
europepmc   +1 more source

The predawn dilemma in adeno-associated virus-based gene therapies for hereditary deafness. [PDF]

Am J Stem Cells
Zhou J, Deng D, Gan C, Du J, Zhao Y.
europepmc   +1 more source

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Adenoviral Vector-Mediated Expression of Physiologic Levels of Human Factor VIII in Nonhuman Primates

Human Gene Therapy, 1999
An E1-, E2a-, E3-deleted adenoviral vector (Av3H82) encoding an epitope-tagged B domain-deleted human factor VIII cDNA (flagged FVIII) was evaluated in nonhuman primates. Twelve cynomolgus monkeys received intravenous administration of Av3H82; 6 monkeys received 6 x 10(11) particles/kg and another 6 received 3 x 10(12) particles/kg.
Michael Kaleko, Soumitra Roy
exaly   +3 more sources

Comparative Transduction Efficiencies of Human and Nonhuman Adenoviral Vectors in Human, Murine, Bovine, and Porcine Cells in Culture

Biochemical and Biophysical Research Communications, 2005
Clinical usefulness of human Ad serotype 5 (HAd5) based vectors is limited primarily because of preexisting Ad immunity and lack of targeting to specific cell types. Alternative vectors based on less prevalent HAd serotypes as well as nonhuman adenoviruses such as porcine Ad serotype 3 (PAd3) and bovine Ad serotype 3 (BAd3) are being developed to ...
Suresh K Mittal, Dinesh S Bangari
exaly   +3 more sources

Acute Toxicity After High-Dose Systemic Injection of Helper-Dependent Adenoviral Vectors Into Nonhuman Primates

Human Gene Therapy, 2004
Systemic intravascular delivery of adenoviral (Ad) vectors for liver-directed gene therapy has been widely employed because of its simplicity, noninvasiveness, and potential for high transduction. For first-generation Ad vectors (FGAd), this results in high but transient levels of transgene expression and long-term hepatotoxicity due to viral gene ...
Nicola Brunetti-Pierri, Arthur L Beaudet, Donna Palmer   +2 more
exaly   +4 more sources

Improved Hepatic Transduction, Reduced Systemic Vector Dissemination, and Long-Term Transgene Expression by Delivering Helper-Dependent Adenoviral Vectors Into the Surgically Isolated Liver of Nonhuman Primates

Human Gene Therapy, 2006
Helper-dependent adenoviral vectors (HDAds) are attractive vectors for liver-directed gene therapy because they can mediate sustained, high-level transgene expression without chronic toxicity. However, high vector doses are required to achieve efficient hepatic transduction by systemic delivery because of a nonlinear dose response.
William Cioffi, MD, Milton J Finegold, Thomas S C Ng   +2 more
exaly   +4 more sources