Výsledky vyhledávání - "Amer F. Saleh"

Akademický článek

Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy

Autor: Alberto Malerba, Jagjeet K Kang, Graham McClorey, Amer F Saleh, Linda Popplewell, Michael J Gait, Matthew JA Wood, George Dickson

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 1, Iss C (2012)

The knockdown of myostatin, a negative regulator of skeletal muscle mass may have important implications in disease conditions accompanied by muscle mass loss like cancer, HIV/AIDS, sarcopenia, muscle atrophy, and Duchenne muscular dystrophy (DMD). I

Externí odkaz: https://doaj.org/article/58a7f59b4ffc4b1299392e488bfee511

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Akademický článek

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

Autor: Corinne Betts, Amer F Saleh, Andrey A Arzumanov, Suzan M Hammond, Caroline Godfrey, Thibault Coursindel, Michael J Gait, Matthew JA Wood

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 1, Iss C (2012)

Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin pre-mRNA splicing, thereby specifically restoring the dystrophin reading frame and generating a

Externí odkaz: https://doaj.org/article/bbb94f06c18043ce89b202330292cdea

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Extracellular vesicles induce minimal hepatotoxicity and immunogenicity

Autor: Olga Shatnyeva, Emilyanne Leonard, Xabier Osteikoetxea, Mick D. Fellows, Amer F. Saleh, Nicholas Edmunds, Madeleine Ingelsten, Elisa Lázaro-Ibáñez, Maryam Clausen, Stephanie M. Bates, Niek Dekker, Fredrik Karlsson, Damla Etal, Nikki Heath, Jonathan A. Rose, Jayne Harris, Maelle Mairesse, Malin Forsgard

Publikováno v: Nanoscale. 11(14)

Extracellular vesicles (EVs) mediate cellular communication through the transfer of active biomolecules, raising interest in using them as biological delivery vehicles for therapeutic drugs. For drug delivery applications, it is important to understa

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b56b3955673a81597a009549e206474c
https://pubmed.ncbi.nlm.nih.gov/30916672

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2'-O-(2-Methoxyethyl) Nucleosides Are Not Phosphorylated or Incorporated Into the Genome of Human Lymphoblastoid TK6 Cells

Autor: Nicholas Edmunds, Amer F. Saleh, Catherine C. Priestley, Martin Bachman, Patrik U. Andersson, Mick D. Fellows, Nigel J. Gooderham, Scott P. Henry

Publikováno v: Toxicological sciences : an official journal of the Society of Toxicology. 163(1)

Nucleoside analogs with 2'-modified sugar moieties are often used to improve the RNA target affinity and nuclease resistance of therapeutic oligonucleotides in preclinical and clinical development. Despite their enhanced nuclease resistance, oligonuc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::826d1f1edc6849c12ecb5c0173e69407
https://pubmed.ncbi.nlm.nih.gov/29325107

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Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

Autor: Ngoc Lu-Nguyen, Amer F. Saleh, George Dickson, Linda Popplewell, Michael J. Gait, Susan Jarmin

Publikováno v: Molecular Therapy. 23:1341-1348

The fatal X-linked Duchenne muscular dystrophy (DMD), characterized by progressive muscle wasting and muscle weakness, is caused by mutations within the DMD gene. The use of antisense oligonucleotides (AOs) modulating pre-mRNA splicing to restore the

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::db1040939da369f2ca63c8d99976811a
https://doi.org/10.1038/mt.2015.88

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Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides

Autor: Amer F. Saleh, Ülo Langel, Mattias Hällbrink, Michael J. Gait, Graham McClorey, Samir El Andaloussi, Suzan M. Hammond, Matthew J.A. Wood, C. I. Edvard Smith, Peter Järver, Andrey A. Arzumanov, Eman M. Zaghloul

Publikováno v: Nucleic Acid Therapeutics

Oligonucleotide analogs have provided novel therapeutics targeting various disorders. However, their poor cellular uptake remains a major obstacle for their clinical development. Negatively charged oligonucleotides, such as 2′-O-Methyl RNA and lock

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a9b4fb2758b64af6424147955c879861
https://doi.org/10.1089/nat.2014.0511

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Re-evaluation of the Mutagenic Response to Phosphorothioate Nucleotides in Human Lymphoblastoid TK6 Cells

Autor: Catherine C. Priestley, Mick D. Fellows, Amer F. Saleh, Nigel J. Gooderham

Publikováno v: Toxicological Sciences. 145:169-176

Oligonucleotide (OND)-based therapeutics and antisense ONDs in particular, are widely used for modulating gene expression inside cells with promising clinical potential (Graham et al., 2013; Thomas et al., 2013). Various chemical modifications to the

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::52d83860147b6890fca6ba4d32ee6747
https://doi.org/10.1093/toxsci/kfv043

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