200 related articles for article (PubMed ID: 37470509)
1. RNAi therapeutics for diseases involving protein aggregation: fazirsiran for alpha-1 antitrypsin deficiency-associated liver disease.
Strnad P; San Martin J
Expert Opin Investig Drugs; 2023; 32(7):571-581. PubMed ID: 37470509
[TBL] [Abstract][Full Text] [Related]
2. Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.
Turner AM; Stolk J; Bals R; Lickliter JD; Hamilton J; Christianson DR; Given BD; Burdon JG; Loomba R; Stoller JK; Teckman JH
J Hepatol; 2018 Aug; 69(2):378-384. PubMed ID: 29572094
[TBL] [Abstract][Full Text] [Related]
3. Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease.
Wooddell CI; Blomenkamp K; Peterson RM; Subbotin VM; Schwabe C; Hamilton J; Chu Q; Christianson DR; Hegge JO; Kolbe J; Hamilton HL; Branca-Afrazi MF; Given BD; Lewis DL; Gane E; Kanner SB; Teckman JH
JCI Insight; 2020 Jun; 5(12):. PubMed ID: 32379724
[TBL] [Abstract][Full Text] [Related]
4. Fazirsiran for Liver Disease Associated with Alpha
Strnad P; Mandorfer M; Choudhury G; Griffiths W; Trautwein C; Loomba R; Schluep T; Chang T; Yi M; Given BD; Hamilton JC; San Martin J; Teckman JH
N Engl J Med; 2022 Aug; 387(6):514-524. PubMed ID: 35748699
[TBL] [Abstract][Full Text] [Related]
5. Hepatocyte proteomes reveal the role of protein disulfide isomerase 4 in alpha 1-antitrypsin deficiency.
Karatas E; Raymond AA; Leon C; Dupuy JW; Di-Tommaso S; Senant N; Collardeau-Frachon S; Ruiz M; Lachaux A; Saltel F; Bouchecareilh M
JHEP Rep; 2021 Aug; 3(4):100297. PubMed ID: 34151245
[TBL] [Abstract][Full Text] [Related]
6. Cleaning up alpha-1 antitrypsin deficiency related liver disease.
Rademacher L; Fromme M; Strnad P
Curr Opin Gastroenterol; 2023 May; 39(3):163-168. PubMed ID: 37144533
[TBL] [Abstract][Full Text] [Related]
7. Alpha-1 antitrypsin deficiency: current therapy and emerging targets.
McElvaney OF; Fraughen DD; McElvaney OJ; Carroll TP; McElvaney NG
Expert Rev Respir Med; 2023 Mar; 17(3):191-202. PubMed ID: 36896570
[TBL] [Abstract][Full Text] [Related]
8. The Autophagy Pathway: A Critical Route in the Disposal of Alpha 1-Antitrypsin Aggregates That Holds Many Mysteries.
Leon C; Bouchecareilh M
Int J Mol Sci; 2021 Feb; 22(4):. PubMed ID: 33668611
[TBL] [Abstract][Full Text] [Related]
9. Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice.
Shen S; Sanchez ME; Blomenkamp K; Corcoran EM; Marco E; Yudkoff CJ; Jiang H; Teckman JH; Bumcrot D; Albright CF
Hum Gene Ther; 2018 Aug; 29(8):861-873. PubMed ID: 29641323
[TBL] [Abstract][Full Text] [Related]
10. Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin-related liver disease in mice.
Guo S; Booten SL; Aghajan M; Hung G; Zhao C; Blomenkamp K; Gattis D; Watt A; Freier SM; Teckman JH; McCaleb ML; Monia BP
J Clin Invest; 2014 Jan; 124(1):251-61. PubMed ID: 24355919
[TBL] [Abstract][Full Text] [Related]
11. Gene therapy for alpha-1 antitrypsin deficiency: an update.
Pires Ferreira D; Gruntman AM; Flotte TR
Expert Opin Biol Ther; 2023 Mar; 23(3):283-291. PubMed ID: 36825473
[TBL] [Abstract][Full Text] [Related]
12. Recent advances in understanding and treating COPD related to α
Henao MP; Craig TJ
Expert Rev Respir Med; 2016 Dec; 10(12):1281-1294. PubMed ID: 27771979
[TBL] [Abstract][Full Text] [Related]
13. In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.
Cruz PE; Mueller C; Cossette TL; Golant A; Tang Q; Beattie SG; Brantly M; Campbell-Thompson M; Blomenkamp KS; Teckman JH; Flotte TR
Lab Invest; 2007 Sep; 87(9):893-902. PubMed ID: 17592477
[TBL] [Abstract][Full Text] [Related]
14. Pediatric and Adult Liver Disease in Alpha-1 Antitrypsin Deficiency.
Ruiz M; Lacaille F; Schrader C; Pons M; Socha P; Krag A; Sturm E; Bouchecareilh M; Strnad P
Semin Liver Dis; 2023 Aug; 43(3):258-266. PubMed ID: 37402396
[TBL] [Abstract][Full Text] [Related]
15. Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease.
Guo S; Booten SL; Watt A; Alvarado L; Freier SM; Teckman JH; McCaleb ML; Monia BP
Rare Dis; 2014; 2():e28511. PubMed ID: 25054094
[TBL] [Abstract][Full Text] [Related]
16. Alpha-1 antitrypsin deficiency: A re-surfacing adult liver disorder.
Fromme M; Schneider CV; Trautwein C; Brunetti-Pierri N; Strnad P
J Hepatol; 2022 Apr; 76(4):946-958. PubMed ID: 34848258
[TBL] [Abstract][Full Text] [Related]
17. Protein modeling to assess the pathogenicity of rare variants of SERPINA1 in patients suspected of having Alpha 1 Antitrypsin Deficiency.
Kueppers F; Andrake MD; Xu Q; Dunbrack RL; Kim J; Sanders CL
BMC Med Genet; 2019 Jul; 20(1):125. PubMed ID: 31307431
[TBL] [Abstract][Full Text] [Related]
18. Knockdown of Z Mutant Alpha-1 Antitrypsin In Vivo Using Modified DNA Antisense Oligonucleotides.
Aghajan M; Guo S; Monia BP
Methods Mol Biol; 2017; 1639():127-138. PubMed ID: 28752452
[TBL] [Abstract][Full Text] [Related]
19. The Mechanism of Mitochondrial Injury in Alpha-1 Antitrypsin Deficiency Mediated Liver Disease.
Khodayari N; Wang RL; Oshins R; Lu Y; Millett M; Aranyos AM; Mostofizadeh S; Scindia Y; Flagg TO; Brantly M
Int J Mol Sci; 2021 Dec; 22(24):. PubMed ID: 34948056
[TBL] [Abstract][Full Text] [Related]
20. Quantitative Lipid Profiling Reveals Major Differences between Liver Organoids with Normal Pi*M and Deficient Pi*Z Variants of Alpha-1-antitrypsin.
Pérez-Luz S; Lalchandani J; Matamala N; Barrero MJ; Gil-Martín S; Saz SR; Varona S; Monzón S; Cuesta I; Justo I; Marcacuzco A; Hierro L; Garfia C; Gomez-Mariano G; Janciauskiene S; Martínez-Delgado B
Int J Mol Sci; 2023 Aug; 24(15):. PubMed ID: 37569847
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
