Zobrazeno 1 - 10
of 18
pro vyhledávání: '"J. Robert Dorkin"'
Autor:
Elena M. Smekalova, Maria G. Martinez, Emmanuel Combe, Anuj Kumar, Selam Dejene, Dominique Leboeuf, Chao-Ying Chen, J. Robert Dorkin, Lan Shuan Shuang, Sarah Kieft, Lauren Young, Luis Alberto Barrera, Michael S. Packer, Giuseppe Ciaramella, Barbara Testoni, Francine Gregoire, Fabien Zoulim
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102112- (2024)
Chronic hepatitis B virus (HBV) infection remains a global health problem due to the lack of treatments that prevent viral rebound from HBV covalently closed circular (ccc)DNA. In addition, HBV DNA integrates in the human genome, serving as a source
Externí odkaz:
https://doaj.org/article/079b4123912c4274a76ea24a7da66556
Autor:
Kevin J. Kauffman, Matthias A. Oberli, J. Robert Dorkin, Juan E. Hurtado, James C. Kaczmarek, Shivani Bhadani, Jeff Wyckoff, Robert Langer, Ana Jaklenec, Daniel G. Anderson
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 10, Iss , Pp 55-63 (2018)
mRNA therapeutics hold promise for the treatment of diseases requiring intracellular protein expression and for use in genome editing systems, but mRNA must transfect the desired tissue and cell type to be efficacious. Nanoparticle vectors that deliv
Externí odkaz:
https://doaj.org/article/e39d3fa298a14a7eb9f00028a6eb6d73
Autor:
Mark W. Tibbitt, Rebecca L. McClellan, J. Robert Dorkin, Robert Langer, Frank Derosa, Owen S. Fenton, Eric A. Appel, Kevin J. Kauffman, Daniel G. Anderson, Michael W. Heartlein
Publikováno v:
PMC
Thousands of human diseases could be treated by selectively controlling the expression of specific proteins in vivo. A new series of alkenyl amino alcohol (AAA) ionizable lipid nanoparticles (LNPs) capable of delivering human mRNA with unprecedented
Autor:
J. Robert Dorkin, Frank Derosa, Daniel G. Anderson, Michael W. Heartlein, Philip H. Chang, Dganit Danino, Yizhou Dong, Benjamin C. Tang, Weiheng Wang, Matthew J. Webber, Junghoon Yang, Robert Langer, Inbal Abutbul-Ionita
Publikováno v:
Nano Letters. 16:842-848
Safe and effective delivery is required for siRNA and mRNA-based therapeutics to reach their potential. Here, we report on the development of poly(glycoamidoamine) brush nanoparticles as delivery vehicles for siRNA and mRNA. These polymers were capab
Autor:
Rosemary Lynn Kanasty, Daniel G. Anderson, Arturo J. Vegas, Ahmed A. Eltoukhy, Hao Yin, J. Robert Dorkin
Publikováno v:
Nature Reviews Genetics. 15:541-555
Gene-based therapy is the intentional modulation of gene expression in specific cells to treat pathological conditions. This modulation is accomplished by introducing exogenous nucleic acids such as DNA, mRNA, small interfering RNA (siRNA), microRNA
Autor:
Christopher A. Alabi, Tatiana Novobrantseva, Roman L. Bogorad, William Querbes, Weiheng Wang, Abigail K. R. Lytton-Jean, Daniel G. Anderson, Avi Schroeder, Akin Akinc, Arturo J. Vegas, Hao Yin, Carmen Barnes, Yi Chen, Delai Chen, Kevin Fitzgerald, J. Robert Dorkin, June Qin, Scott A Barros, Victor Koteliansky, Yizhou Dong, Robert Langer, Julia Hettinger, Sasilada Sirirungruang, Gaurav Sahay, Kevin T. Love, Daniel J. Siegwart, Mary Carioto, Varun Kumar, Yunlong Zhang, Karsten Olejnik
Publikováno v:
Proceedings of the National Academy of Sciences. 111:3955-3960
siRNA therapeutics have promise for the treatment of a wide range of genetic disorders. Motivated by lipoproteins, we report lipopeptide nanoparticles as potent and selective siRNA carriers with a wide therapeutic index. Lead material cKK-E12 showed
Autor:
Ana Jaklenec, Matthias A. Oberli, Michael J. Mitchell, Owen S. Fenton, Andreas M. Reichmuth, Daniel G. Anderson, J. Robert Dorkin, Daniel Blankschtein, Robert Langer
The induction of a strong cytotoxic T cell response is an important prerequisite for successful immunotherapy against many viral diseases and tumors. Nucleotide vaccines, including mRNA vaccines with their intracellular antigen synthesis, have been s
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0abe77dd6e3788a27288f9d615f59506
https://europepmc.org/articles/PMC5523404/
https://europepmc.org/articles/PMC5523404/
Autor:
William Querbes, Muthiah Manoharan, K. Narayanannair Jayaprakash, Mark A Tracy, Kallanthottathil G. Rajeev, Dinah W.Y. Sah, Michael J. Hope, Antonin de Fougerolles, Victor Koteliansky, Soma De, Akin Akinc, Eugenio Fava, June Qin, Andrew Sprague, Anja Zeigerer, Martin Maier, Liuliang Qin, James Butler, William Cantley, Kevin Fitzgerald, J. Robert Dorkin, Marino Zerial, Muthusamy Jayaraman, Maria Frank-Kamenetsky, Timothy Racie
Publikováno v:
Molecular Therapy. 18(7):1357-1364
Lipid nanoparticles (LNPs) have proven to be highly efficient carriers of short-interfering RNAs (siRNAs) to hepatocytes in vivo; however, the precise mechanism by which this efficient delivery occurs has yet to be elucidated. We found that apolipopr
Autor:
Ammen P. Sandhu, Scott A Barros, William Cantley, Ying K. Tam, Masuna Srinivasulu, Verbena Kosovrasti, Akin Akinc, Kieu Lam, Kallanthottathil G. Rajeev, Marco A. Ciufolini, Dinah W.Y. Sah, Soma De, Lloyd Jeffs, Ismail M. Hafez, Qingmin Chen, Thomas D. Madden, Antonin de Fougerolles, Ed Yaworski, June Qin, Mark A Tracy, Pieter R. Cullis, Barbara L. Mui, Jianxin Chen, Michael J. Hope, Martin Maier, Sandra K. Klimuk, Rene Alvarez, Connie K Cho, Michael J Weinstein, Muthiah Manoharan, Kim F. Wong, Mikameh Kazem, Derrick Stebbing, Merete L. Eisenhardt, Ian MacLachlan, Lubomir Nechev, Erin J Crosley, Muthusamy Jayaraman, J. Robert Dorkin, Sean C. Semple, Todd Borland
Publikováno v:
Nature Biotechnology. 28:172-176
We adopted a rational approach to design cationic lipids for use in formulations to deliver small interfering RNA (siRNA). Starting with the ionizable cationic lipid 1,2-dilinoleyloxy-3-dimethylaminopropane (DLinDMA), a key lipid component of stable
Autor:
Jung H. Yang, Owen S. Fenton, J. Robert Dorkin, Kevin J. Kauffman, Michael W. Heartlein, Frank Derosa, Faryal F. Mir, Daniel G. Anderson
Publikováno v:
Nano letters. 15(11)
Intracellular delivery of messenger RNA (mRNA) has the potential to induce protein production for many therapeutic applications. Although lipid nanoparticles have shown considerable promise for the delivery of small interfering RNAs (siRNA), their ut