Results 21 to 30 of about 89,643 (298)
Generation of enhanced gene delivery vectors by directed evolution of adeno-associated virus [PDF]
Despite promising advance in the development of viral vectors based on AAV for human gene therapy, several major hurdles for a more general use remain.
Endell, Jan
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A novel dual mechanism-of-action bispecific PD-1-IL-2v armed by a “βγ-only” interleukin-2 variant
IntroductionInterleukin-2 (IL-2) is one of the first cytokines to be discovered as an immune agonist for cancer immunotherapy. Biased IL-2 variants had been discovered to eliminate Treg activation or enhance the tumor specific T cell cytotoxicity ...
Yongji Jiang +7 more
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Characterization of Brevibacillus laterosporus Cas9 (BlatCas9) for Mammalian Genome Editing
Compact CRISPR/Cas9 systems that can be delivered by AAV for in vivo genome editing hold great promise for clinical applications. Brevibacillus laterosporus Cas9 (BlatCas9) is a compact Cas9 nuclease that has been identified for plant genome editing ...
Ning Gao +8 more
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PEP-TP56 enhances AAV transduction in a serotype-independent manner
Adeno-associated viruses (AAVs) have emerged as the most prominent gene therapy vectors. However, the need for high AAV doses to achieve clinical efficacy remains a key challenge to AAV utility in the clinic.
Olaniyi Olarewaju +4 more
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Real-time PCR analysis of RNA obtained from the nodules treated with AAV overexpressing Tis21 (AAV-Tis21) or AAV-CBA (control). Three nodules per group were analyzed. Average ± SEM values are from (at least) three independent experiments and are shown as
Manuela Ceccarelli (4398151) +9 more
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Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We?
Divya Ail, Hugo Malki, Emilia A Zin, Deniz Dalkara Sorbonne Université, INSERM, CNRS, Department of Therapeutics, Institut de la Vision, Paris, 75012, FranceCorrespondence: Divya Ail, Department of Therapeutics, Institut de la Vision, 17 Rue Moreau ...
Ail D, Malki H, Zin EA, Dalkara D
doaj
AAV-mediated overexpression of Cytl1 induces CF.
Control virus or AAV-Cytl1 (5 × 1010 viral genome) was injected into the tail vein of WT mice, and the phenotype of the heart was examined after 8 wks. (A) Picrosirius staining of heart cross-sections from WT mice injected with control virus or AAV-Cytl.
Roger J. Hajjar (142214) +8 more
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Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors [PDF]
BACKGROUND: Inactivating genes in vivo is an important technique for establishing their function in the adult nervous system. Unfortunately, conventional knockout mice may suffer from several limitations including embryonic or perinatal lethality and the
Hermens, Wim T J M C +37 more
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Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations [PDF]
The unique palindromic inverted terminal repeats (ITRs) and single-stranded nature of adeno-associated virus (AAV) DNA are major hurdles to current sequencing technologies.
Roland W. Herzog +15 more
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Techniques to genetically manipulate the activity of defined neuronal subpopulations have been useful in elucidating function, however applicability to translational research beyond transgenic mice is limited. Subtype targeted transgene expression can be
Heinrich S Gompf +4 more
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