Results 71 to 80 of about 182,997 (357)

Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA‐Charge Neutral Linkages for Enhanced Splice‐Switching Antisense Oligonucleotides

Angewandte Chemie, EarlyView.
Splice‐switching oligonucleotides are used to treat severe genetic conditions including Duchenne Muscular Dystrophy, but new chemistries are urgently required to improve their efficacy. In this work locked nucleic acid (LNA) is coupled to different charge neutral DNA backbones which are studied by structural, thermodynamic, and biological methods.
Alice Kennett, Lillian Lie, Martin Flerin, Belma Zengin Kurt, Ysobel R. Baker, Alyssa C. Hill, Abinaya Ramesh, Matthew J.A. Wood, Debashis Dhara, Afaf H. El‐Sagheer, Fernanda Duarte, Tom Brown   +11 more
wiley   +2 more sources

CDK inhibitors for muscle stem cell differentiation and self-renewal

Journal of Physical Fitness and Sports Medicine, 2017
Regeneration of muscle is undertaken by muscle stem cell populations named satellite cells which are normally quiescent or at the G0 phase of the cell cycle.
Amrudha Mohan, Atsushi Asakura
doaj   +1 more source

Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism

Cell Death and Disease, 2023
Facioscapulohumeral muscular dystrophy (FSHD) is among the most common of the muscular dystrophies, affecting nearly 1 in 8000 individuals, and is a cause of profound disability.
Justin Cohen, Shushu Huang, Katherine E. Koczwara, Kristen T. Woods, Vincent Ho, Keryn G. Woodman, Jack L. Arbiser, Katelyn Daman, Monkol Lek, Charles P. Emerson, Alec M. DeSimone   +10 more
doaj   +1 more source

Genetic modifiers of ambulation in the cooperative international Neuromuscular Research Group Duchenne natural history study [PDF]

, 2015
OBJECTIVE: We studied the effects of LTBP4 and SPP1 polymorphisms on age at loss of ambulation (LoA) in a multiethnic Duchenne muscular dystrophy (DMD) cohort.
Andreone, Luz, Bello, Luca, Cnaan, Avital, Cooperative International Neuromuscular Research Group Investigators, Dubrovsky, Alberto, Duong, Tina, Gordish Dressman, Heather, Henricson, Erik K., Hoffman, Eric P., Kesari, Akanchha, McDonald, Craig M., Morgenroth, Lauren P., Pegoraro, Elena, Punetha, Jaya   +13 more
core   +1 more source

Anticodon Engineered Transfer RNA (tRNASUAG) Inhibits Hepatitis B Virus Replication by Promoting the Degradation of Core Protein

Advanced Science, EarlyView.
This study employed tRNASUAG, an anticodon‐engineered tRNA (ACE‐tRNA), to introduce a phosphorylatable serine into the C‐terminal domain of HBc by reading through its stop codon, thereby potentially promoting the ubiquitin‐proteasome degradation of HBc, and subsequently inhibiting HBV replication. Through a tRNASUAG‐gHBV1‐tRNASUAG‐gHBV2‐tRNASUAG tandem
Xingwen Yang, Huiying Sun, Ziheng Luo, Qinxin Zhang, Yarong Song, Jie Li, Xiaoyun Liu, Jie Wang   +7 more
wiley   +1 more source

A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing

Molecular Therapy: Nucleic Acids, 2022
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which disrupt the dystrophin open reading frame (ORF), represent one of the most common types ...
Yu Zhang, Hui Li, Takahiko Nishiyama, John R. McAnally, Efrain Sanchez-Ortiz, Jian Huang, Pradeep P.A. Mammen, Rhonda Bassel-Duby, Eric N. Olson   +8 more
doaj   +1 more source

Facioscapulohumeral Muscular Dystrophy

Neurotherapeutics, 2004
Facioscapulohumeral muscular dystrophy (FSHD), a dominantly inherited disorder, is the third most common dystrophy after Duchenne and myotonic muscular dystrophy. No known effective treatments exist for FSHD. The lack of an understanding of the underlying pathophysiology remains an obstacle in the development of targeted therapeutic interventions.
openaire   +8 more sources

TREM2‐Mediated Cholesterol Efflux in Macrophages Inhibits Anti‐Tumor Immunity via Limitation of CD4+ T and NK Cells

Advanced Science, EarlyView.
Impaired anti‐tumor immunity is mediated by TREM2 + macrophages in non‐small cell lung cancer. TREM2 + macrophages prevent anti‐tumor immunity depending on the limitation of CD4 + T and NK cells. TREM2 promotes cholesterol efflux via ABCA1 to limit the production of CX3CL1 in macrophages.
Yunhan Wang, Weina Yu, Xinxin Wang, Qitai Zhao, Qingyang Lei, Aitian Li, Shasha Liu, Tian Wang, Li Yang, Yi Zhang   +9 more
wiley   +1 more source

Lipids of dystrophic and normal mouse muscle: whole tissue and particulate fractions

Journal of Lipid Research, 1970
Myofibrillar, mitochondrial, and microsomal fractions were prepared from normal and dystrophic mouse limb muscle by differential centrifugation and analyzed for phospholipids and cholesterol.
K. OWENS, B.P. HUGHES
doaj   +1 more source

Are mice good models for human neuromuscular disease? Comparing muscle excursions in walking between mice and humans [PDF]

, 2017
The mouse is one of the most widely used animal models to study neuromuscular diseases and test new therapeutic strategies. However, findings from successful pre-clinical studies using mouse models frequently fail to translate to humans due to various ...
A De Luca, A Rajagopal, A Soman, AA Biewener, AA Biewener, AC Ludolph, AEH Emery, AL Camp, AV Birn-Jeffery, BH Wokke, BJ Petrof, C Pastoret, C Webster, CM Consolino, CM Eng, DM Escolar, E Azizi, E Mok, EK Moo, EM Arnold, EM Arnold, EM Arnold, FA Jenkins, FE Zajac, GM Buyse, GT Carter, H Radley-Crabb, H Stedman, HB van der Worp, HM Hsieh-Li, IG Kirkinezos, J Manning, JA Granchelli, JA Vilensky, JH Veldink, JJ Kaczor, JK Mah, JN Kornegay, JN Kornegay, JP Charles, JP Charles, JR Chamberlain, JW Carnwath, JW McGreevy, KA Clarke, KN An, L Xu, LM Ittner, M Benatar, M Bodor, M Millard, MD Grounds, MD Klein Horsman, ME Gurney, ME Llewellyn, MP Kadaba, N Alexander, N Serradj, NP Whitehead, P DeVita, P Moens, P Sicinski, R Burdi, R Willmann, R Willmann, RH Miller, RW Burgess, SL Delp, SL Delp, SR Ward, SS Blemker, SV Brooks, T Akay, TA McMahon, TA Partridge, TS Buchanan, V Brussee, VC Henderson, X Chen, X Chen, X Hu, ZJ Domire   +81 more
core   +2 more sources