260 related articles for article (PubMed ID: 36345805)
1. Liver-directed drugs for transthyretin-mediated amyloidosis.
Brannagan TH; Berk JL; Gillmore JD; Maurer MS; Waddington-Cruz M; Fontana M; Masri A; Obici L; Brambatti M; Baker BF; Hannan LA; Buchele G; Viney NJ; Coelho T; Nativi-Nicolau J
J Peripher Nerv Syst; 2022 Dec; 27(4):228-237. PubMed ID: 36345805
[TBL] [Abstract][Full Text] [Related]
2. Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis.
Butler JS; Chan A; Costelha S; Fishman S; Willoughby JL; Borland TD; Milstein S; Foster DJ; Gonçalves P; Chen Q; Qin J; Bettencourt BR; Sah DW; Alvarez R; Rajeev KG; Manoharan M; Fitzgerald K; Meyers RE; Nochur SV; Saraiva MJ; Zimmermann TS
Amyloid; 2016 Jun; 23(2):109-18. PubMed ID: 27033334
[TBL] [Abstract][Full Text] [Related]
3. RNA-targeting and gene editing therapies for transthyretin amyloidosis.
Aimo A; Castiglione V; Rapezzi C; Franzini M; Panichella G; Vergaro G; Gillmore J; Fontana M; Passino C; Emdin M
Nat Rev Cardiol; 2022 Oct; 19(10):655-667. PubMed ID: 35322226
[TBL] [Abstract][Full Text] [Related]
4. Recent Advances in Oligonucleotide-Based Therapy for Transthyretin Amyloidosis: Clinical Impact and Future Prospects.
Hayashi Y; Jono H
Biol Pharm Bull; 2018; 41(12):1737-1744. PubMed ID: 30504675
[TBL] [Abstract][Full Text] [Related]
5. RNA Targeting and Gene Editing Strategies for Transthyretin Amyloidosis.
Ioannou A; Fontana M; Gillmore JD
BioDrugs; 2023 Mar; 37(2):127-142. PubMed ID: 36795354
[TBL] [Abstract][Full Text] [Related]
6. Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments.
Sekijima Y
J Neurol Neurosurg Psychiatry; 2015 Sep; 86(9):1036-43. PubMed ID: 25604431
[TBL] [Abstract][Full Text] [Related]
7. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
Gillmore JD; Gane E; Taubel J; Kao J; Fontana M; Maitland ML; Seitzer J; O'Connell D; Walsh KR; Wood K; Phillips J; Xu Y; Amaral A; Boyd AP; Cehelsky JE; McKee MD; Schiermeier A; Harari O; Murphy A; Kyratsous CA; Zambrowicz B; Soltys R; Gutstein DE; Leonard J; Sepp-Lorenzino L; Lebwohl D
N Engl J Med; 2021 Aug; 385(6):493-502. PubMed ID: 34215024
[TBL] [Abstract][Full Text] [Related]
8. Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran.
Titze-de-Almeida SS; Brandão PRP; Faber I; Titze-de-Almeida R
Mol Diagn Ther; 2020 Feb; 24(1):49-59. PubMed ID: 31701435
[TBL] [Abstract][Full Text] [Related]
9. Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data.
Viney NJ; Guo S; Tai LJ; Baker BF; Aghajan M; Jung SW; Yu RZ; Booten S; Murray H; Machemer T; Burel S; Murray S; Buchele G; Tsimikas S; Schneider E; Geary RS; Benson MD; Monia BP
ESC Heart Fail; 2021 Feb; 8(1):652-661. PubMed ID: 33283485
[TBL] [Abstract][Full Text] [Related]
10. Hepatocyte-Targeted Delivery of siRNA Polyplex with PEG-Modified Lactosylated Dendrimer/Cyclodextrin Conjugates for Transthyretin-Related Amyloidosis Therapy.
Hayashi Y; Higashi T; Motoyama K; Jono H; Ando Y; Onodera R; Arima H
Biol Pharm Bull; 2019; 42(10):1679-1688. PubMed ID: 31582656
[TBL] [Abstract][Full Text] [Related]
11. Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides.
Ackermann EJ; Guo S; Benson MD; Booten S; Freier S; Hughes SG; Kim TW; Jesse Kwoh T; Matson J; Norris D; Yu R; Watt A; Monia BP
Amyloid; 2016 Sep; 23(3):148-157. PubMed ID: 27355239
[TBL] [Abstract][Full Text] [Related]
12. APOE polymorphism in ATTR amyloidosis patients treated with lipid nanoparticle siRNA.
Niemietz C; Nadzemova O; Zibert A; Schmidt HH
Amyloid; 2020 Mar; 27(1):45-51. PubMed ID: 31648569
[TBL] [Abstract][Full Text] [Related]
13. Tafamidis: a selective transthyretin stabilizer to treat wild-type ATTR amyloidosis and hereditary ATTR amyloidosis with cardiomyopathy.
Paton DM
Drugs Today (Barc); 2019 Dec; 55(12):727-734. PubMed ID: 31942875
[TBL] [Abstract][Full Text] [Related]
14. A Review of Novel Agents and Clinical Considerations in Patients With ATTR Cardiac Amyloidosis.
Benbrahim M; Norman K; Sanchorawala V; Siddiqi OK; Hughes D
J Cardiovasc Pharmacol; 2021 May; 77(5):544-548. PubMed ID: 33657048
[TBL] [Abstract][Full Text] [Related]
15. Feasibility Study of Dendrimer-Based TTR-CRISPR pDNA Polyplex for Ocular Amyloidosis in Vitro.
Inoue M; Muta K; Mohammed AFA; Onodera R; Higashi T; Ouchi K; Ueda M; Ando Y; Arima H; Jono H; Motoyama K
Biol Pharm Bull; 2022; 45(11):1660-1668. PubMed ID: 36328502
[TBL] [Abstract][Full Text] [Related]
16. Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis.
Niemietz C; Chandhok G; Schmidt H
Molecules; 2015 Sep; 20(10):17944-75. PubMed ID: 26437390
[TBL] [Abstract][Full Text] [Related]
17. Selective silencing of a mutant transthyretin allele by small interfering RNAs.
Kurosawa T; Igarashi S; Nishizawa M; Onodera O
Biochem Biophys Res Commun; 2005 Nov; 337(3):1012-8. PubMed ID: 16225852
[TBL] [Abstract][Full Text] [Related]
18. Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases.
Greig JA; Breton C; Ashley SN; Martins KM; Gorsuch C; Chorazeczewski JK; Furmanak T; Smith MK; Zhu Y; Bell P; Shoop W; Li H; Smith J; Tomberlin G; Clark P; Mitchell TW; Buza EL; Yan H; Jantz D; Wilson JM
Hum Gene Ther; 2022 Nov; 33(21-22):1174-1186. PubMed ID: 36375122
[TBL] [Abstract][Full Text] [Related]
19. Tolcapone, a potent aggregation inhibitor for the treatment of familial leptomeningeal amyloidosis.
Pinheiro F; Varejão N; Esperante S; Santos J; Velázquez-Campoy A; Reverter D; Pallarès I; Ventura S
FEBS J; 2021 Jan; 288(1):310-324. PubMed ID: 32324953
[TBL] [Abstract][Full Text] [Related]
20. Patisiran for the treatment of patients with familial amyloid polyneuropathy.
Rizk M; Tüzmen S
Drugs Today (Barc); 2019 May; 55(5):315-327. PubMed ID: 31131842
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
