Results 21 to 30 of about 9,370,992 (242)

Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors [PDF]

, 2004
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, Lieberman, A., Eggers, R., Verhaagen, J., Sablitzky, Fred, Chakravarthy, Sridhara, Zhang, Jing Ying, Zhang, J.Y., Hermens, Wim TJMC, Zhang, Jing, Verhaagen, J, Ahmed, Bushra, Anderson, P.N., Eggers, R, Eggers, Ruben, Levelt, Christiaan, Chakravarthy, S, Levelt, C., Ahmed, BY, Verhaagen, Joost, Lieberman, A.R., Hermens, WTJMC, Lieberman, AR, Anderson, Patrick N., Ahmed, Bushra Y, Hermens, W.T., Niclou, SP, Anderson, PN, NICLOU, Simone P., Lieberman, A R, Zhang, JY, Anderson, Patrick N, Sablitzky, F., Levelt, C, Sablitzky, F, Niclou, S.P., Chakravarthy, S., Ahmed, B.Y.   +37 more
core   +1 more source

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice [PDF]

, 2009
Background: Adeno-associated virus serotype 2 (AAV2) vectors show considerable promise for ocular gene transfer. However, one potential barrier to efficacious long-term therapy is the development of immune responses against the vector or transgene ...
Ali, R.R., Barker, S.E., Robbie, S.J., Smith, A.J., Natkunarajah, M., Buch, P., Balaggan, K.S., Duran, Y., MacLaren, R.E., Bainbridge, J.W. B., Broderick, C.A.   +10 more
core   +1 more source

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

Molecular Therapy: Methods & Clinical Development, 2019
Adeno-associated virus (AAV) capsid libraries have generated improved transgene delivery vectors. We designed an AAV library construct, iTransduce, that combines a peptide library on the AAV9 capsid with a Cre cassette to enable sensitive detection of ...
Killian S. Hanlon, Jonah C. Meltzer, Tetyana Buzhdygan, Ming J. Cheng, Miguel Sena-Esteves, Rachel E. Bennett, Timothy P. Sullivan, Roshanak Razmpour, Yi Gong, Carrie Ng, Josette Nammour, Daniela Maiz, Simon Dujardin, Servio H. Ramirez, Eloise Hudry, Casey A. Maguire   +15 more
doaj   +1 more source

Perinatal Gene Transfer to the Liver [PDF]

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Ward, N.J., Buckley, S.M.K., Ward, NJ, Chan, J.K.Y., Waddington, S.N., Rahim, A.A., Waddington, SN, Rahim, AA, Chan, JK, Mckay, T.R., Howe, S.J., Howe, SJ, McKay, TR, Natalie J. Ward, Ahad A. Rahim, Steven J. Howe, Jerry K.Y.Chan, Suzanne M.K. Buckley, Simon N. Waddington, Tristan R. McKay   +20 more
core   +1 more source

Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors

Molecular Therapy: Methods & Clinical Development, 2023
Based on the observation that humans have variable responses of gene expression with the same dose of an adeno-associated vector, we hypothesized that there are deleterious variants in genes coding for processes required for adeno-associated virus (AAV ...
Mahboubeh R. Rostami, Philip L. Leopold, Jenifer M. Vasquez, Miguel de Mulder Rougvie, Alya Al Shakaki, Ali Ait Hssain, Amal Robay, Neil R. Hackett, Jason G. Mezey, Ronald G. Crystal   +9 more
doaj   +1 more source

Process Improvement of Adeno-Associated Virus Production [PDF]

Frontiers in Chemical Engineering, 2022
Adeno-associated viruses (AAVs) have been well characterized and used to deliver therapeutic genes for diseases treatment in clinics and basic research. This study used the triple transient transfection of AAV-DJ/8 as a model expression system to develop and optimize the laboratory production of AAV for research and pre-clinical applications ...
Jia-Shiung Guan, Kai Chen, Yingnan Si, Taehyun Kim, Zhuoxin Zhou, Seulhee Kim, Lufang Zhou, Xiaoguang Liu   +7 more
openaire   +3 more sources

AAV vectors accumulate in the pineal gland after injections into the brain or spinal cord

Molecular Therapy: Methods & Clinical Development, 2021
AAV vectors are being used extensively for gene-modifying therapies for neurological disorders. Here, we report the surprising discovery that injections of different AAVs into the brain, spinal cord, or cerebrospinal fluid (CSF) lead to robust ...
Oswald Steward, Aminata P. Coulibaly, Mariajose Metcalfe, Jamie M. Dam, Kelly M. Yee   +4 more
doaj   +1 more source

Generation of enhanced gene delivery vectors by directed evolution of adeno-associated virus [PDF]

, 2006
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
core   +1 more source

In Vivo Genetic Manipulation of Spermatogonial Stem Cells and Their Microenvironment by Adeno-Associated Viruses

Stem Cell Reports, 2018
Summary: Adeno-associated virus (AAV) penetrates the blood-brain barrier, but it is unknown whether AAV penetrates other tight junctions. Genetic manipulation of testis has been hampered by the basement membrane of seminiferous tubules and the blood ...
Satoshi Watanabe, Mito Kanatsu-Shinohara, Narumi Ogonuki, Shogo Matoba, Atsuo Ogura, Takashi Shinohara   +5 more
doaj   +1 more source

AAV genome modification for efficient AAV production

Heliyon, 2023
The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a ...
Walaa Asaad, Polina Volos, Denis Maksimov, Elena Khavina, Andrei Deviatkin, Olga Mityaeva, Pavel Volchkov   +6 more
doaj   +1 more source