Results 41 to 50 of about 144,727 (384)

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

BioDrugs, 2017
There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells ...
M. Naso, B. Tomkowicz, William L Perry, W. Strohl   +3 more
semanticscholar   +1 more source

The E3 ubiquitin ligase IDOL regulates synaptic ApoER2 levels and is important for plasticity and learning. [PDF]

, 2017
Neuronal ApoE receptors are linked to learning and memory, but the pathways governing their abundance, and the mechanisms by which they affect the function of neural circuits are incompletely understood.
Achiro, Jennifer M, Choi, Jinkuk, Gao, Jie, Kim, Sangmok, Li, Sandy, Marosi, Mate, Martin, Kelsey C, Otis, Klara, Portera-Cailliau, Carlos, Tontonoz, Peter   +9 more
core   +3 more sources

Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease [PDF]

, 2009
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease resulting from the expansion of a glutamine repeat in the huntingtin (Htt) protein.
Ko, Jan, Patterson, Paul H., Southwell, Amber L.   +2 more
core   +2 more sources

Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

Nature Communications, 2020
Adeno-associated virus (AAV) forms the basis for several commercial gene therapy products and for countless gene transfer vectors derived from natural or synthetic viral isolates that are under intense preclinical evaluation.
J. Weinmann, S. Weis, Josefine Sippel, W. Tulalamba, A. Remes, Jihad El Andari, A. Herrmann, Q. H. Pham, Christopher Borowski, S. Hille, T. Schönberger, N. Frey, M. Lenter, T. VandenDriessche, O. Müller, M. Chuah, T. Lamla, D. Grimm   +17 more
semanticscholar   +1 more source

Characterization of Brevibacillus laterosporus Cas9 (BlatCas9) for Mammalian Genome Editing

Frontiers in Cell and Developmental Biology, 2020
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, Ning Gao, Chengdong Zhang, Ziying Hu, Miaomiao Li, Jingjing Wei, Yongming Wang, Yongming Wang, Huihui Liu   +8 more
doaj   +1 more source

Rabies screen reveals GPe control of cocaine-triggered plasticity. [PDF]

, 2017
Identification of neural circuit changes that contribute to behavioural plasticity has routinely been conducted on candidate circuits that were preselected on the basis of previous results.
AE Stepien, AH Gittis, B Weissbourd, BN Armbruster, Boris D. Heifets, C Hammond, C Lüscher, Christina K. Kim, CM Bäckman, D Lin, EJ Nestler, ES Boyden, F-M Zhou, G Schiavo, H Taniguchi, HH Yin, IR Wickersham, JA Shuen, JS Park, K Miyamichi, K Miyamichi, K Yonehara, Karl Deisseroth, Katherine E. DeLoach, Kevin T. Beier, KF Raab-Graham, KT Beier, L Madisen, L Petreanu, L Ye, LA Gunaydin, LA Schwarz, Lin Wai Hung, Liqun Luo, M Watabe-Uchida, M Xue, MJ Krashes, NR Wall, P Rajasethupathy, Paul Hoerbelt, PE Rothwell, PW Kalivas, RC Pierce, RL Albin, Robert C. Malenka, S Hippenmeyer, S Lammel, S Lammel, SHR Oliet, Sophie Neuner, Timothy J. Mosca, TJ Mosca, TJ Stachniak, TN Lerner, Y Smith   +54 more
core   +2 more sources

A novel dual mechanism-of-action bispecific PD-1-IL-2v armed by a “βγ-only” interleukin-2 variant

Frontiers in Immunology
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, Chuyuan Chen, Yuan Liu, Rong Wang, Chuan Feng, Lili Cai, Shuang Chang, Lei Zhao   +7 more
doaj   +1 more source

Plectin-1 targeted AAV vector for the molecular imaging of pancreatic cancer

Frontiers in Oncology, 2013
Pancreatic ductal adenocarcinoma (PDAC) is highly malignant disease that is the 4th leading cause of cancer-related death in the US. Gene therapy using AAV vectors to selectively deliver genes to PDAC cells is an attractive treatment option for ...
Prasad R. Konkalmatt, Defeng eDeng, Stephanie eThomas, Michael T. Wu, Craig D. Logsdon, Brent A. French, Kimberly A Kelly   +6 more
doaj   +1 more source

AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness

Advancement of science
Mutations in OTOFERLIN (OTOF) lead to the autosomal recessive deafness 9 (DFNB9). The efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is extensively validated in Otof‐deficient mice.
J. Qi, Fangzhi Tan, Liyan Zhang, Ling Lu, Shanzhong Zhang, Yabo Zhai, Yicheng Lu, X. Qian, Wenxiu Dong, Yinyi Zhou, Ziyu Zhang, Xuehan Yang, Lulu Jiang, Chaorong Yu, Jiancheng Liu, Tian Chen, Lianqiu Wu, Chang Tan, Sijie Sun, Huaien Song, Y. Shu, Lei Xu, Xia Gao, Huawei Li, Renjie Chai   +24 more
semanticscholar   +1 more source

LRH-1 mitigates intestinal inflammatory disease by maintaining epithelial homeostasis and cell survival. [PDF]

, 2018
Epithelial dysfunction and crypt destruction are defining features of inflammatory bowel disease (IBD). However, current IBD therapies targeting epithelial dysfunction are lacking.
Bayrer, James R, Escusa, Hazel S, Fletterick, Robert J, Ingraham, Holly A, Klein, Ophir D, Moore, David D, Nattiv, Roy, Suzawa, Miyuki, Wang, Hongtao   +8 more
core   +2 more sources