Zobrazeno 1 - 10
of 995
pro vyhledávání: '"A. Khvorova"'
Autor:
James W. Gilbert, Zachary Kennedy, Bruno M.D.C. Godinho, Ashley Summers, Alexandra Weiss, Dimas Echeverria, Brianna Bramato, Nicholas McHugh, David Cooper, Ken Yamada, Matthew Hassler, Hélène Tran, Fen Biao Gao, Robert H. Brown, Jr., Anastasia Khvorova
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 3, Pp 102291- (2024)
A hexanucleotide (G4C2) repeat expansion (HRE) within intron one of C9ORF72 is the leading genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). C9ORF72 haploinsufficiency, formation of RNA foci, and production of di
Externí odkaz:
https://doaj.org/article/48f420ec0d5f4364889fb47b75a6618c
Autor:
Rachael Miller, Joseph Paquette, Alexandra Barker, Ellen Sapp, Nicholas McHugh, Brianna Bramato, Nozomi Yamada, Julia Alterman, Dimas Echeveria, Ken Yamada, Jonathan Watts, Christelle Anaclet, Marian DiFiglia, Anastasia Khvorova, Neil Aronin
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102359- (2024)
Oligonucleotide therapeutics (ASOs and siRNAs) have been explored for modulation of gene expression in the central nervous system (CNS), with several drugs approved and many in clinical evaluation. Administration of highly concentrated oligonucleotid
Externí odkaz:
https://doaj.org/article/7a3e9e86c8024f13a243cc537165a446
Autor:
Vignesh N. Hariharan, Takahiro Nakamura, Minwook Shin, Qi Tang, Vyankat Sontakke, Jillian Caiazzi, Samuel Hildebrand, Anastasia Khvorova, Ken Yamada
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 2, Pp 102230- (2024)
Small interfering RNAs (siRNAs) are revolutionizing the treatment of liver-associated indications. Yet, robust delivery to extrahepatic tissues remains a challenge. Conjugating lipids (e.g., docosanoic acid [DCA]) to siRNA supports extrahepatic deliv
Externí odkaz:
https://doaj.org/article/11b7081549e04a32bb58409f85966f34
Autor:
Gilbert, James W., Kennedy, Zachary, Godinho, Bruno M.D.C., Summers, Ashley, Weiss, Alexandra, Echeverria, Dimas, Bramato, Brianna, McHugh, Nicholas, Cooper, David, Yamada, Ken, Hassler, Matthew, Tran, Hélène, Gao, Fen Biao, Brown, Robert H., Jr., Khvorova, Anastasia
Publikováno v:
In Molecular Therapy - Nucleic Acids 10 September 2024 35(3)
Autor:
Miller, Rachael, Paquette, Joseph, Barker, Alexandra, Sapp, Ellen, McHugh, Nicholas, Bramato, Brianna, Yamada, Nozomi, Alterman, Julia, Echeveria, Dimas, Yamada, Ken, Watts, Jonathan, Anaclet, Christelle, DiFiglia, Marian, Khvorova, Anastasia, Aronin, Neil
Publikováno v:
In Molecular Therapy - Nucleic Acids 10 December 2024 35(4)
Autor:
Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Hui Wang, Nicholas McHugh, Brianna Bramato, Brett Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
Publikováno v:
eLife, Vol 12 (2024)
Circulating lactate is a fuel source for liver metabolism but may exacerbate metabolic diseases such as nonalcoholic steatohepatitis (NASH). Indeed, haploinsufficiency of lactate transporter monocarboxylate transporter 1 (MCT1) in mice reportedly pro
Externí odkaz:
https://doaj.org/article/874400b9307a469685643153d134f013
Autor:
Qi Tang, Katherine Y. Gross, Hassan H. Fakih, Samuel O. Jackson, Mohammad Zain U.I. Abideen, Kathryn R. Monopoli, Carine Blanchard, Claire Bouix-Peter, Thibaud Portal, John E. Harris, Anastasia Khvorova, Julia F. Alterman
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102117- (2024)
Identifying therapeutic oligonucleotides that are cross-reactive to experimental animal species can dramatically accelerate the process of preclinical development and clinical translation. Here, we identify fully chemically-modified small interfering
Externí odkaz:
https://doaj.org/article/20b9e2482d6e42e2ba4c20b806a408b4
Autor:
Shun-Yun Cheng, Jillian Caiazzi, Annabelle Biscans, Julia F. Alterman, Dimas Echeverria, Nicholas McHugh, Matthew Hassler, Samson Jolly, Delaney Giguere, Joris Cipi, Anastasia Khvorova, Claudio Punzo
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102088- (2024)
Inherited retinal dystrophies caused by dominant mutations in photoreceptor (PR) cell expressed genes are a major cause of irreversible vision loss. Oligonucleotide therapy has been of interest in diseases that conventional medicine cannot target. In
Externí odkaz:
https://doaj.org/article/345e01d43abe4fa98e498b15c9d879c2
Autor:
Qi Tang, Hassan H. Fakih, Mohammad Zain UI Abideen, Samuel R. Hildebrand, Khashayar Afshari, Katherine Y. Gross, Jacquelyn Sousa, Allison S. Maebius, Christina Bartholdy, Pia Pernille Søgaard, Malene Jackerott, Vignesh Hariharan, Ashley Summers, Xueli Fan, Ken Okamura, Kathryn R. Monopoli, David A. Cooper, Dimas Echeverria, Brianna Bramato, Nicholas McHugh, Raymond C. Furgal, Karen Dresser, Sarah J. Winter, Annabelle Biscans, Jane Chuprin, Nazgol-Sadat Haddadi, Shany Sherman, Ümmügülsüm Yıldız-Altay, Mehdi Rashighi, Jillian M. Richmond, Claire Bouix-Peter, Carine Blanchard, Adam Clauss, Julia F. Alterman, Anastasia Khvorova, John E. Harris
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-15 (2023)
Abstract Inhibition of Janus kinase (JAK) family enzymes is a popular strategy for treating inflammatory and autoimmune skin diseases. In the clinic, small molecule JAK inhibitors show distinct efficacy and safety profiles, likely reflecting variable
Externí odkaz:
https://doaj.org/article/4a8cb6635b9d4fb98e9074b6bcb3b50f
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 33, Iss , Pp 93-109 (2023)
Chemically modified small interfering RNAs (siRNAs) are promising therapeutics guiding sequence-specific silencing of disease genes. Identifying chemically modified siRNA sequences that effectively silence target genes remains challenging. Such deter
Externí odkaz:
https://doaj.org/article/53d9349f29c8424096fc0c8893af6f24