Zobrazeno 1 - 10
of 16
pro vyhledávání: '"Laura Adamson-Small"'
Autor:
Chady H. Hakim, Nathalie Clément, Lakmini P. Wasala, Hsiao T. Yang, Yongping Yue, Keqing Zhang, Kasun Kodippili, Laura Adamson-Small, Xiufang Pan, Joel S. Schneider, N. Nora Yang, Jeffrey S. Chamberlain, Barry J. Byrne, Dongsheng Duan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 664-678 (2020)
Vector production scale-up is a major barrier in systemic adeno-associated virus (AAV) gene therapy. Many scalable manufacturing methods have been developed. However, the potency of the vectors generated by these methods has rarely been compared with
Externí odkaz:
https://doaj.org/article/c9a8a753dbc949b9af502c5c8edc3335
Autor:
Seemin S. Ahmed, Hillard Rubin, Minglun Wang, Deiby Faulkner, Arnold Sengooba, Serena N. Dollive, Nancy Avila, Jeff L. Ellsworth, Diana Lamppu, Maria Lobikin, Jason Lotterhand, Laura Adamson-Small, Teresa Wright, Albert Seymour, Omar L. Francone
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 568-580 (2020)
Phenylketonuria is an inborn error of metabolism caused by loss of function of the liver-expressed enzyme phenylalanine hydroxylase and is characterized by elevated systemic phenylalanine levels that are neurotoxic. Current therapies do not address t
Externí odkaz:
https://doaj.org/article/b01d454355934bc48f5e9e66ef3b1092
Autor:
Hazel C Levy, Danielle Hulvey, Laura Adamson-Small, Natacha Jn-Simon, Victor Prima, Scott Rivkees, Jacqueline A Hobbs
Publikováno v:
PLoS ONE, Vol 15, Iss 2, p e0228005 (2020)
Targeted gene therapy using recombinant adeno-associated virus (rAAV) vectors is a potential therapeutic strategy for treating cancer, and tissue-specific promoters may help with tissue targeting. Medullary thyroid carcinoma (MTC) is a disease of the
Externí odkaz:
https://doaj.org/article/30f8a00c966c4fcfb5360940bc435566
Autor:
Huei-Mei Chen, Rachel Resendes, Azita Ghodssi, Danielle Sookiasian, Michael Tian, Serena Dollive, Laura Adamson-Small, Nancy Avila, Cagdas Tazearslan, John F Thompson, Jeff L Ellsworth, Omar Francone, Albert Seymour, Jason B Wright
Publikováno v:
PLoS ONE, Vol 15, Iss 5, p e0233373 (2020)
Targeted gene integration via precise homologous recombination (HR)-based gene editing has the potential to correct genetic diseases. AAV (adeno-associated virus) can mediate nuclease-free gene integration at a disease-causing locus. Therapeutic appl
Externí odkaz:
https://doaj.org/article/f8a88c5666bb403d9b0cc2f575ccb6c7
Autor:
Laura Adamson-Small, Mark Potter, Darin J Falk, Brian Cleaver, Barry J Byrne, Nathalie Clément
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Recombinant adeno-associated vectors based on serotype 9 (rAAV9) have demonstrated highly effective gene transfer in multiple animal models of muscular dystrophies and other neurological indications. Current limitations in vector production and purif
Externí odkaz:
https://doaj.org/article/4d765c4b74044eacb94a800c8510d23f
Publikováno v:
Cell and Gene Therapy Insights. 6:1249-1262
Autor:
Dongsheng Duan, Yongping Yue, Lakmini P. Wasala, Xiufang Pan, Jeffrey S. Chamberlain, N. Nora Yang, Chady H. Hakim, Nathalie Clement, Joel S. Schneider, Keqing Zhang, Barry J. Byrne, Kasun Kodippili, Laura Adamson-Small, Hsiao T. Yang
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 664-678 (2020)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Vector production scale-up is a major barrier in systemic adeno-associated virus (AAV) gene therapy. Many scalable manufacturing methods have been developed. However, the potency of the vectors generated by these methods has rarely been compared with
Autor:
Minglun Wang, Hillard Rubin, Jason Lotterhand, Omar L. Francone, Maria Lobikin, Seemin Seher Ahmed, Albert B. Seymour, Diana Lamppu, Laura Adamson-Small, Serena Nicole Dollive, Jeff L. Ellsworth, Teresa Leah Wright, Nancy Avila, Arnold Sengooba, Deiby Faulkner
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 568-580 (2020)
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 568-580 (2020)
Phenylketonuria is an inborn error of metabolism caused by loss of function of the liver-expressed enzyme phenylalanine hydroxylase and is characterized by elevated systemic phenylalanine levels that are neurotoxic. Current therapies do not address t
Autor:
Natacha Jn-Simon, Hazel C. Levy, Scott A. Rivkees, Danielle Hulvey, Laura Adamson-Small, Victor Prima, Jacqueline A. Hobbs
Publikováno v:
PLoS ONE, Vol 15, Iss 2, p e0228005 (2020)
PLoS ONE
PLoS ONE
Targeted gene therapy using recombinant adeno-associated virus (rAAV) vectors is a potential therapeutic strategy for treating cancer, and tissue-specific promoters may help with tissue targeting. Medullary thyroid carcinoma (MTC) is a disease of the
Publikováno v:
Human Gene Therapy Methods
The increase in effective treatments using recombinant adeno-associated viral (rAAV) vectors has underscored the importance of scalable, high-yield manufacturing methods. Previous work from this group reported the use of recombinant herpes simplex vi