Zobrazeno 1 - 10
of 16
pro vyhledávání: '"Zachary Kennedy"'
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:
Qing Xie, Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang, Zachary Kennedy, Alisha Gruntman, Ailing Du, Qin Su, Ran He, Phillip WL Tai, Guangping Gao, Jun Xie
Publikováno v:
EMBO Molecular Medicine, Vol 16, Iss 4, Pp 945-965 (2024)
Abstract Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), how
Externí odkaz:
https://doaj.org/article/4e7a21bfc0d24ec095ce22f8d8fa7575
Autor:
Bruno M.D.C. Godinho, Emily G. Knox, Samuel Hildebrand, James W. Gilbert, Dimas Echeverria, Zachary Kennedy, Reka A. Haraszti, Chantal M. Ferguson, Andrew H. Coles, Annabelle Biscans, Jillian Caiazzi, Julia F. Alterman, Matthew R. Hassler, Anastasia Khvorova
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 29, Iss , Pp 116-132 (2022)
Effective systemic delivery of small interfering RNAs (siRNAs) to tissues other than liver remains a challenge. siRNAs are small (∼15 kDa) and therefore rapidly cleared by the kidneys, resulting in limited blood residence times and tissue exposure.
Externí odkaz:
https://doaj.org/article/085fd4bc93584da8ae49dde6a3e10019
Autor:
Haiwei Mou, Deniz M. Ozata, Jordan L. Smith, Ankur Sheel, Suet-Yan Kwan, Soren Hough, Alper Kucukural, Zachary Kennedy, Yueying Cao, Wen Xue
Publikováno v:
Genome Medicine, Vol 11, Iss 1, Pp 1-11 (2019)
Abstract CRISPR/Cas9 has revolutionized cancer mouse models. Although loss-of-function genetics by CRISPR/Cas9 is well-established, generating gain-of-function alleles in somatic cancer models is still challenging because of the low efficiency of gen
Externí odkaz:
https://doaj.org/article/578de3f1d83f4486ba462701b06c2cc8
Autor:
Daniel O'Reilly, Jillian Belgrad, Chantal Ferguson, Ashley Summers, Ellen Sapp, Cassandra McHugh, Ella Mathews, Adel Boudi, Julianna Buchwald, Socheata Ly, Dimas Moreno, Raymond Furgal, Eric Luu, Zachary Kennedy, Vignesh Hariharan, Kathryn Monopoli, X. William Yang, Jeffery Carroll, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Publikováno v:
Molecular Therapy.
Autor:
Vignesh N. Hariharan, Minwook Shin, Ching-Wen Chang, Daniel O’Reilly, Annabelle Biscans, Ken Yamada, Zhiru Guo, Mohan Somasundaran, Qi Tang, Kathryn Monopoli, Pranathi Meda Krishnamurthy, Gitali Devi, Nicholas McHugh, David A. Cooper, Dimas Echeverria, John Cruz, Io Long Chan, Ping Liu, Sun-Young Lim, Jill McConnell, Satya Prakash Singh, Samuel Hildebrand, Jacquelyn Sousa, Sarah M. Davis, Zachary Kennedy, Chantal Ferguson, Bruno M. D. C. Godinho, Yann Thillier, Jillian Caiazzi, Socheata Ly, Manish Muhuri, Karen Kelly, Fiachra Humphries, Alyssa Cousineau, Krishna Mohan Parsi, Qi Li, Yang Wang, René Maehr, Guangping Gao, Dmitry Korkin, William M. McDougall, Robert W. Finberg, Katherine A. Fitzgerald, Jennifer P. Wang, Jonathan K. Watts, Anastasia Khvorova
Publikováno v:
Proceedings of the National Academy of Sciences. 120
The continuous evolution of SARS-CoV-2 variants complicates efforts to combat the ongoing pandemic, underscoring the need for a dynamic platform for the rapid development of pan-viral variant therapeutics. Oligonucleotide therapeutics are enhancing t
Autor:
Daniel O’Reilly, Jillian Belgrad, Chantal Ferguson, Ashley Summers, Ellen Sapp, Cassandra McHugh, Ella Mathews, Julianna Buchwald, Socheata Ly, Dimas Echeverria Moreno, Zachary Kennedy, Vignesh Hariharan, Kathryn Monopoli, X. William Yang, Jeffery Carroll, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Huntington’s Disease (HD) is a severe neurodegenerative disorder caused by expansion of the CAG trinucleotide repeat tract in the huntingtin gene. Inheritance of expanded CAG repeats is needed for HD manifestation, but further somatic expansion of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::b85abae4738303ac45e081a5dee520ab
https://doi.org/10.1101/2022.09.06.506795
https://doi.org/10.1101/2022.09.06.506795
Publikováno v:
Methods Mol Biol
Methods in Molecular Biology ISBN: 9781071620090
Methods in Molecular Biology ISBN: 9781071620090
Therapeutic oligonucleotides hold tremendous potential for treating central nervous system (CNS) disorders. The route of administration of oligonucleotides significantly impacts both distribution and silencing efficiency. Here, we describe a technica
Autor:
Pin‐Tsun Justin Lee, Zachary Kennedy, Yuzhen Wang, Yimeng Lu, Carolina Cefaliello, Özgün Uyan, Chun‐Qing Song, Bruno Miguel da Cruz Godinho, Zuoshang Xu, Mary Rusckowski, Wen Xue, Robert H. Brown
Publikováno v:
Annals of neurology. 91(5)
The objective of this study is to develop a novel method for monitoring the integrity of motor neurons in vivo by quantifying net retrograde axonal transport.The method uses single photon emission computed tomography to quantify retrograde transport
Autor:
Bruno M.D.C. Godinho, Emily G. Knox, Samuel Hildebrand, James W. Gilbert, Dimas Echeverria, Zachary Kennedy, Reka A. Haraszti, Chantal M. Ferguson, Andrew H. Coles, Annabelle Biscans, Jillian Caiazzi, Julia F. Alterman, Matthew R. Hassler, Anastasia Khvorova
Publikováno v:
Molecular therapy. Nucleic acids. 29
Effective systemic delivery of small interfering RNAs (siRNAs) to tissues other than liver remains a challenge. siRNAs are small (∼15 kDa) and therefore rapidly cleared by the kidneys, resulting in limited blood residence times and tissue exposure.