Results 51 to 60 of about 9,649,206 (293)

AAV-based vectors for human diseases modeling in laboratory animals

Frontiers in Medicine
The development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models.
Timur I. Aliev, Dmitry V. Yudkin
doaj   +1 more source

Transduction of rat pancreatic islets with pseudotyped adeno-associated virus vectors

Virology Journal, 2009
Background Pancreatic islet transplantation is a promising treatment for type I diabetes mellitus, but current immunosuppressive strategies do not consistently provide long-term survival of transplanted islets.
Blanchette-Mackie E Joan, Chiorini John A, McAlister Victor J, Schmidt Michael, Pechhold Klaus, Liu Eric, Pack Stephanie, Jou William, Dwyer Nancy K, Gavrilova Oksana, Craig Anthony T, Harlan David M, Owens Roland A   +12 more
doaj   +1 more source

Disrupted TFEB/GDNF–cAMP/ATP Coupling Underlies Astrocytic Dysfunction and Depression in LRRK2 G2019S Parkinson's Mice

Advanced Science, EarlyView.
ABSTRACT The LRRK2 G2019S mutation, a Parkinson's disease–linked variant, has been associated with depression‐like phenotypes, but mechanisms remain unclear. We chart age‐dependent behavioral changes and astrocyte reactivity in G2019S mice and define a pathway connecting LRRK2 to TFEB/GDNF signaling, cellular energetics, and inflammation.
Longping Yao, Maryam Hatami, Sumeyye Koc, Qing Jiang, Thomas Skutella, Yuanfeng Zhang   +5 more
wiley   +1 more source

An improved high-resolution method for quantitative separation of empty and filled AAV8 capsids by strong anion exchange HPLC

Frontiers in Bioengineering and Biotechnology
Cell and gene therapy (CGT) is a field of therapeutic medicine that aims to treat, prevent, and cure diseases using engineered cells (stem cells, immune cells, and differentiated adult or fetal cells), vectors [Adeno Associated Virus (AAV), Adeno Virus ...
Samantha Schrecke, Kevin McManus, Cassandra Moshfegh, Jessica Stone, Thuy-Uyen Nguyen, Gustavo Rivas, Ismaeel Muhamed, Daniel A. J. Mitchell   +7 more
doaj   +1 more source

AAV2.7m8 is a powerful viral vector for inner ear gene therapy

Nature Communications, 2019
Adeno-associated virus is used in gene therapy in mouse models of hearing loss. Here the authors compare vectors and find AAV2.7m8 can infect cells in the inner ear with high efficiency.
Kevin Isgrig, Devin S. McDougald, Jianliang Zhu, Hong Jun Wang, Jean Bennett, Wade W. Chien   +5 more
doaj   +1 more source

Preclinical Assessment of Immune Responses to AAV (adeno-associated virus) Vectors

Frontiers in Immunology, 2014
Transitioning to human trials from preclinical models resulted in the emergence of inhibitory AAV vector immune responses which has become a hurdle for sustained correction.
Etiena eBasner-Tschakarjan, Enoch eBijjiga, Ashley Thomas Martino   +2 more
doaj   +1 more source

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]

, 2003
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Spence, Y, Ng, CYC, Nathwani, AC, Davidoff, AM, Zhou, J   +4 more
core  

Viral Hybrid Vectors for Somatic Integration - Are They the Better Solution? [PDF]

, 2009
The turbulent history of clinical trials in viral gene therapy has taught us important lessons about vector design and safety issues. Much effort was spent on analyzing genotoxicity after somatic integration of therapeutic DNA into the host genome. Based
Anja Ehrhardt, Nadine Müther, Ehrhardt, Anja, Nadja Noske, Müther, Nadine, Noske, Nadja, Nadine Müther   +6 more
core   +1 more source

Pre‐Regenerative Endothelial Cells Empowered by Antler Blood Exosomes Orchestrate Peripheral Nerve Repair

Advanced Science, EarlyView.
The pre‐regenerative vascular niche (PVN) is essential for nerve repair, yet its endothelial blueprint remains unclear. We identified angiogenic ECs (AECs) as the dominant pre‐regenerative subset and found that antler blood–derived exosomes (AB‐EXO) promote repair via IMP3.
Jinsheng Huang, Qiang Chen, Aihua Jin, Xiaoqi Yang, Hui Zhou, Weiquan Gong, Jin Cheng, Jun Dai, Minjin Wang, Meihua Piao, Yuanyi Wang, Nan Zhou   +11 more
wiley   +1 more source

When size matters: A novel compact Cas12a variant for in vivo genome editing.

PLoS Biology
A new study characterizes and improves a novel small Cas12a variant before adapting it for in vivo genome editing by delivery via adeno-associated virus (AAV) vectors, showcasing the potential of small CRISPR systems and their compatibility with viral ...
Felix Bubeck, Dirk Grimm
doaj   +1 more source