Results 101 to 110 of about 396,665 (375)

Circumventing antivector immunity: potential use of nonhuman adenoviral vectors [PDF]

, 2014
Adenoviruses are efficient gene delivery vectors based on their ability to transduce a wide variety of cell types and drive high-level transient transgene expression. While there have been advances in modifying human adenoviral (HAdV) vectors to increase
Beard C.W., Benkö M., Cauthen A.N., Crawford-Miksza L., Estrella Lopez-Gordo, Gall J., Gall J.G., Gattoni A., Ginsberg H.S., Haviv Y.S., Hofmann C., Iva I. Podgorski, Kajon A.E., Kanerva A., Kass-Eisler A., Kuriyama S., Mastrangeli A., Michou A.I., Nicholas Downes, Ramon Alemany, Rogozhin V.N., Roy S., Seiradake E., Shmarov M.M., Soudais C., Tutykhina I.L., Wang D.Y., Wohlfart C., Ying B., Zakhartchouk A.N., Zeng Q.   +30 more
core   +3 more sources

Adeno-associated virus for cystic fibrosis gene therapy

Brazilian Journal of Medical and Biological Research, 2011
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by ...
S.V. Martini, P.R.M. Rocco, M.M. Morales
doaj   +3 more sources

Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation

bioRxiv, 2019
Cell-type-specific expression of molecular tools and sensors is critical to construct circuit diagrams and to investigate the activity and function of neurons within the nervous system.
Leila Haery, Benjamin E. Deverman, K. Matho, Ali H. Cetin, K. Woodard, C. Cepko, K. Guerin, Meghan A. Rego, Ina Ersing, Susanna M. Bachle, J. Kamens, Melina Fan   +11 more
semanticscholar   +1 more source

The Adeno-Associated Virus Genome Packaging Puzzle [PDF]

Journal of Molecular and Genetic Medicine, 2015
Chen Ling1,2, Yuan Wang1-3, Yuan Lu4, Lina Wang1-3, Giridhara R Jayandharan5, George V Aslanidi1,2, Baozheng Li1,2, Binbin Cheng1,3, Wenqin Ma1,2, Thomas Lentz6, Changquan Ling3, Xiao Xiao6,7, R Jude Samulski6, Nicholas Muzyczka2,8,9 and Arun Srivastava1,2,8-10* 1Division of Cellular and Molecular Therapy, Department of Pediatrics, University of ...
Yuan Lu, Chen Ling, Thomas B. Lentz, Yuan Wang, Changquan Ling, Nicholas Muzyczka, Xiao Xiao, Lina Wang, Baozheng Li, Binbin Cheng, Giridhara R. Jayandharan, George Aslanidi, R. Jude Samulski, Arun Srivastava, Wenqin Ma   +14 more
openaire   +3 more sources

Pinoresinol Diglucoside Attenuates Nuclear Receptor Coactivator 4‐Mediated Ferritinophagy Associated with Cisplatin‐Induced Hearing Loss

Advanced Science, EarlyView.
Pinoresinol diglucoside, a bioactive component extracted from traditional Chinese medicine, attenuates cisplatin‐induced ototoxicity in vivo and in vitro. The protective effect is achieved by downregulation of Socs1. The present study is the first to investigate the expression and function of Socs1 in inner ear, which is known to promote ferroptosis ...
Yin Chen, Cheng Cheng, Ao Li, Dengbin Ma, Siyu Li, Handong Wang, Song Gao, Dingding Liu, Panpan Song, Chenjie Yu, Xiaoyun Qian, Guoqiang Wan, Xia Gao   +12 more
wiley   +1 more source

Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape

Neural Regeneration Research
Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive
Courtney J Rouse, Victoria N Jensen, Coy D Heldermon   +2 more
doaj   +1 more source

AAVR Expression is Essential for AAV Vector Transduction in Sensory Hair Cells

Advanced Science, EarlyView.
Decreased sensitivity to AAV vector transduction in the outer hair cells (OHCs) of adult mice is primarily attributed to reduction of AAVR (Kiaa0319l; Au040320). Knockout of AAVR reduces AAV vector transduction efficiency in both inner hair cells (IHCs) and OHCs in neonatal mice.
Fan Wu, Guisheng Chen, Rui Hu, Peiwen Liu, Jintao Lou, Wenji Zhao, Zuhong He, Suhua Sha, Yiqing Zheng   +8 more
wiley   +1 more source

Increased risk for T cell autoreactivity to ß-cell antigens in the mice expressing the Avy obesity-associated gene. [PDF]

, 2019
There has been considerable debate as to whether obesity can act as an accelerator of type 1 diabetes (T1D). We assessed this possibility using transgenic mice (MIP-TF mice) whose ß-cells express enhanced green fluorescent protein (EGFP). Infecting these
Atkinson, Mark A, Dang, Hoa, Kaufman, Daniel L, Sun, Ren, Tian, Jide, Wu, Ting-Ting, Yong, Jing   +6 more
core   +2 more sources

Single Administration of AAV‐mAtp6v1b2 Gene Therapy Rescues Hearing and Vestibular Disorders Caused by Atp6v1b2‐Induced Lysosomal Dysfunction in Hair Cells

Advanced Science, EarlyView.
Wei et al. establish a hair cell‐specific conditional knockout mouse model (Atp6v1b2fl/fl;Atoh1Cre/+), and demonstrate the importance of Atp6v1b2 for hair cell through maintaining the survival of lysosomes. A single administration of AAV‐ie‐Eh3‐mAtp6v1b2 through scala media at P0‐P2 realizes function compensation and restores hearing and balance ...
Gege Wei, Shiwei Qiu, Xue Gao, Lu Zheng, Yijin Chen, Ying Ma, Haifeng Feng, Jinyuan Yang, Guojie Dong, Huiyi Nie, Weihao Zhao, Xiaoge Li, Guangqin Wang, Wei Xiong, Pu Dai, Yongyi Yuan   +15 more
wiley   +1 more source

Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates. [PDF]

, 2020
Retinal gene therapy using adeno-associated viruses (AAVs) is constrained by the mode of viral vector delivery. Intravitreal AAV injections are impeded by the internal limiting membrane barrier, while subretinal injections require invasive surgery and ...
Chung, Sook Hyun, Mollhoff, Iris N, Nguyen, Uyen Tu, Noronha, Glenn, Taraborelli, Donna, Thomasy, Sara M, Yiu, Glenn, Yoo, Jesse   +7 more
core