Anastasia Khvorova
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Explore the profile of Anastasia Khvorova including associated specialties, affiliations and a list of published articles.
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Articles
139
Citations
13085
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Recent Articles
1.
Sapp E, Boudi A, Iwanowicz A, Belgrad J, Miller R, OReilly D, et al.
bioRxiv
. 2025 Jan;
PMID: 39803497
has been identified in human and mouse HD brain as the pathogenic exon 1 mRNA generated from aberrant splicing between exon 1 and 2 that contributes to aggregate formation and...
2.
Mathews E, Coffey S, Gartner A, Belgrad J, Bragg R, OReilly D, et al.
bioRxiv
. 2024 Nov;
PMID: 39574582
Huntington's disease (HD) arises from a CAG expansion in the () gene beyond a critical threshold. A major thrust of current HD therapeutic development is lowering levels of mutant mRNA...
3.
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...
4.
Gilbert J, Kennedy Z, Godinho B, Summers A, Weiss A, Echeverria D, et al.
Mol Ther Nucleic Acids
. 2024 Sep;
35(3):102291.
PMID: 39233852
A hexanucleotide (GC) repeat expansion (HRE) within intron one of is the leading genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). haploinsufficiency, formation of RNA foci, and...
5.
Rivera Flores I, Monopoli K, Jackson S, Echeverria D, OReilly D, Brown R, et al.
Nucleic Acid Ther
. 2024 Aug;
34(5):234-244.
PMID: 39189114
Small interfering RNAs (siRNAs) represent a novel class of drugs capable of potent and sustained modulation of genes across various tissues. Preclinical development of siRNAs necessitates assessing efficacy and toxicity...
6.
Yamada K, Hariharan V, Caiazzi J, Miller R, Ferguson C, Sapp E, et al.
Nat Biotechnol
. 2024 Aug;
PMID: 39090305
Therapeutic small interfering RNA (siRNA) requires sugar and backbone modifications to inhibit nuclease degradation. However, metabolic stabilization by phosphorothioate (PS), the only backbone chemistry used clinically, may be insufficient for...
7.
Kloske C, Belloy M, Blue E, Bowman G, Carrillo M, Chen X, et al.
Alzheimers Dement
. 2024 Jul;
20(9):6590-6605.
PMID: 39031528
Introduction: The apolipoprotein E gene (APOE) is an established central player in the pathogenesis of Alzheimer's disease (AD), with distinct apoE isoforms exerting diverse effects. apoE influences not only amyloid-beta...
8.
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...
9.
Susorov D, Echeverria D, Khvorova A, Korostelev A
Nucleic Acids Res
. 2024 Jul;
52(15):8687-8701.
PMID: 39011883
Nonsense mutations account for >10% of human genetic disorders, including cystic fibrosis, Alagille syndrome, and Duchenne muscular dystrophy. A nonsense mutation results in the expression of a truncated protein, and...
10.
Weiss A, Gilbert J, Rivera Flores I, Belgrad J, Ferguson C, Dogan E, et al.
bioRxiv
. 2024 Jul;
PMID: 38979291
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative condition, with 20% of familial and 2-3% of sporadic cases linked to mutations in the cytosolic superoxide dismutase (SOD1) gene. Mutant SOD1...