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Joseph Paquette

Explore the profile of Joseph Paquette including associated specialties, affiliations and a list of published articles. Areas
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Articles 6
Citations 1
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Recent Articles
1.
Miller R, Paquette J, Barker A, Sapp E, McHugh N, Bramato B, et al.
Mol Ther Nucleic Acids . 2024 Nov; 35(4):102359. PMID: 39554992
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...
2.
Allen S, OReilly D, Miller R, Sapp E, Summers A, Paquette J, et al.
Nucleic Acid Ther . 2024 Jul; 34(4):164-172. PMID: 39023561
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by CAG repeat expansion in the first exon of the huntingtin gene (). Oligonucleotide therapeutics, such as short interfering RNA...
3.
Miller R, Paquette J, Barker A, Sapp E, McHugh N, Bramato B, et al.
bioRxiv . 2024 Jun; PMID: 38895198
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...
4.
Allen S, OReilly D, Miller R, Sapp E, Summers A, Paquette J, et al.
bioRxiv . 2024 May; PMID: 38774633
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by CAG repeat expansion in the first exon of the huntingtin gene (). Oligonucleotide therapeutics, such as short interfering RNA...
5.
Hariharan V, Caiazzi J, Miller R, Ferguson C, Sapp E, Fakih H, et al.
Res Sq . 2023 Jul; PMID: 37398145
Metabolic stabilization of therapeutic oligonucleotides requires both sugar and backbone modifications, where phosphorothioate (PS) is the only backbone chemistry used in the clinic. Here, we describe the discovery, synthesis, and...
6.
Yamada K, Hariharan V, Caiazzi J, Miller R, Furguson C, Sapp E, et al.
bioRxiv . 2023 Jun; PMID: 37292886
Metabolic stabilization of therapeutic oligonucleotides requires both sugar and backbone modifications, where phosphorothioate (PS) is the only backbone chemistry used in the clinic. Here, we describe the discovery, synthesis, and...