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pro vyhledávání: '"Luk, H."'
Autor:
Prashant Kumar, Michael Wang, Ozan S. Kumru, John M. Hickey, Julio Sanmiguel, Nerea Zabaleta, Luk H. Vandenberghe, Sangeeta B. Joshi, David B. Volkin
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 103-121 (2023)
Recombinant adeno-associated viruses (rAAVs) are a preferred vector system in clinical gene transfer. A fundamental challenge to formulate and deliver rAAVs as stable and efficacious vaccines is to elucidate interrelationships between the vector’s
Externí odkaz:
https://doaj.org/article/c0cdad5aefc247faaa90ba08588e0e25
Autor:
Ying Hsu, Sajag Bhattarai, Jacob M. Thompson, Angela Mahoney, Jacintha Thomas, Sara K. Mayer, Poppy Datta, Janelle Garrison, Charles C. Searby, Luk H. Vandenberghe, Seongjin Seo, Val C. Sheffield, Arlene V. Drack
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 31, Iss , Pp 164-181 (2023)
Blindness in Bardet-Biedl syndrome (BBS) is caused by dysfunction and loss of photoreceptor cells in the retina. BBS10, mutations of which account for approximately 21% of all BBS cases, encodes a chaperonin protein indispensable for the assembly of
Externí odkaz:
https://doaj.org/article/595390ab39c54dacacc20bd00b9764d5
Autor:
Michael Florea, Fotini Nicolaou, Simon Pacouret, Eric M. Zinn, Julio Sanmiguel, Eva Andres-Mateos, Carmen Unzu, Amy J. Wagers, Luk H. Vandenberghe
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 146-159 (2023)
The adeno-associated viral vector (AAV) provides a safe and efficient gene therapy platform with several approved products that have marked therapeutic impact for patients. However, a major bottleneck in the development and commercialization of AAV r
Externí odkaz:
https://doaj.org/article/b21e4b6154cf46b4a7fae892eb16cad8
Autor:
Kumar, Prashant, Wang, Michael, Kumru, Ozan S., Hickey, John M., Sanmiguel, Julio, Zabaleta, Nerea, Vandenberghe, Luk H., Joshi, Sangeeta B., Volkin, David B.
Publikováno v:
In Molecular Therapy - Methods & Clinical Development 14 September 2023 30:103-121
Autor:
Florea, Michael, Nicolaou, Fotini, Pacouret, Simon, Zinn, Eric M., Sanmiguel, Julio, Andres-Mateos, Eva, Unzu, Carmen, Wagers, Amy J., Vandenberghe, Luk H.
Publikováno v:
In Molecular Therapy - Methods & Clinical Development 9 March 2023 28:146-159
Autor:
Katz, Michael G., Hadas, Yoav, Bailey, Rasheed A., Fazal, Shahood, Vincek, Adam, Madjarova, Sophia J., Shtraizent, Nataly, Vandenberghe, Luk H., Eliyahu, Efrat
Publikováno v:
In The Journal of Thoracic and Cardiovascular Surgery December 2022 164(6):e429-e443
Akademický článek
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Autor:
Zinn, Eric, Unzu, Carmen, Schmit, Pauline F., Turunen, Heikki T., Zabaleta, Nerea, Sanmiguel, Julio, Fieldsend, Allegra, Bhatt, Urja, Diop, Cheikh, Merkel, Erin, Gurrala, Rakesh, Peacker, Bryan, Rios, Christopher, Messemer, Kathleen, Santos, Jennifer, Estelien, Reynette, Andres-Mateos, Eva, Wagers, Amy J., Tipper, Christopher, Vandenberghe, Luk H.
Publikováno v:
In Cell Reports Medicine 15 November 2022 3(11)
Autor:
Eva Andres-Mateos, Lukas D. Landegger, Carmen Unzu, Jean Phillips, Brian M. Lin, Nicholas A. Dewyer, Julio Sanmiguel, Fotini Nicolaou, Michelle D. Valero, Kathrin I. Bourdeu, William F. Sewell, Rudolph J. Beiler, Michael J. McKenna, Konstantina M. Stankovic, Luk H. Vandenberghe
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-10 (2022)
Gene therapy using Adeno-associated viral vectors (AAV) rescues hearing and balance deficits in mouse models of human disorders. Here, the authors show that AAVAnc80L65 allows efficient cochlear gene transfer in nonhuman primates, and motivate future
Externí odkaz:
https://doaj.org/article/7e6b7c587b2a4cc9b15c510a61c21f79
Autor:
Petr O. Ilyinskii, Alicia M. Michaud, Gina L. Rizzo, Christopher J. Roy, Sheldon S. Leung, Stephanie L. Elkins, Teresa Capela, Aparajita Chowdhury, Lina Li, Randy J. Chandler, Irini Manoli, Eva Andres-Mateos, Lloyd P.M. Johnston, Luk H. Vandenberghe, Charles P. Venditti, Takashi Kei Kishimoto
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss , Pp 279-292 (2021)
A major barrier to adeno-associated virus (AAV) gene therapy is the inability to re-dose patients due to formation of vector-induced neutralizing antibodies (Nabs). Tolerogenic nanoparticles encapsulating rapamycin (ImmTOR) provide long-term and spec
Externí odkaz:
https://doaj.org/article/6698961584084f2e8ca2b818f89110a5