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Phase 1 Trials of PNPLA3 siRNA in I148M Homozygous Patients with MAFLD
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2024-08-01 , DOI: 10.1056/nejmc2402341 Elisa Fabbrini 1 , Brian Rady 2 , Anastasiya Koshkina 1 , Jae Yoon Jeon 1 , Vivaswath S. Ayyar 2 , Cynthia Gargano 2 , Nicholas DiProspero 1 , Susan Wendel 1 , Julia Hegge 3 , Holly Hamilton 3 , Zhi-Ming Ding 3 , Maria Afrazi 3 , Anthony Nicholas 3 , Tao Pei 3 , Masayoshi Nakano 4 , Shohei Ouchi 4 , Yuki Saito 4 , Aimi Yamashita 4 , Ryo Tamamura 4 , Hernan Salazar 5 , Craig Shapiro 6 , Tatsuya Yoshihara 7 , Takuma Yonemura 8 , Satoshi Inoue 9 , Osamu Matsuoka 10 , Mark Erion 2 , Alessandro Pocai 2 , Hideo Makimura 1
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2024-08-01 , DOI: 10.1056/nejmc2402341 Elisa Fabbrini 1 , Brian Rady 2 , Anastasiya Koshkina 1 , Jae Yoon Jeon 1 , Vivaswath S. Ayyar 2 , Cynthia Gargano 2 , Nicholas DiProspero 1 , Susan Wendel 1 , Julia Hegge 3 , Holly Hamilton 3 , Zhi-Ming Ding 3 , Maria Afrazi 3 , Anthony Nicholas 3 , Tao Pei 3 , Masayoshi Nakano 4 , Shohei Ouchi 4 , Yuki Saito 4 , Aimi Yamashita 4 , Ryo Tamamura 4 , Hernan Salazar 5 , Craig Shapiro 6 , Tatsuya Yoshihara 7 , Takuma Yonemura 8 , Satoshi Inoue 9 , Osamu Matsuoka 10 , Mark Erion 2 , Alessandro Pocai 2 , Hideo Makimura 1
Affiliation
Homozygosity for the PNPLA3 risk allele is linked to liver fat accumulation. In phase 1 trials, JNJ-75220795, a hepatocyte-targeted GalNAc-conjugated PNPLA3 siRNA, reduced liver fat in PNPLA3 I148M...
中文翻译：
PNPLA3 siRNA 在 I148M 纯合子 MAFLD 患者中的 1 期试验
PNPLA3 风险等位基因的纯合性与肝脏脂肪积累有关。在 1 期试验中，JNJ-75220795（一种针对肝细胞的 GalNAc 缀合的 PNPLA3 siRNA）可减少 PNPLA3 I148M 中的肝脏脂肪...
更新日期：2024-08-02
中文翻译：
PNPLA3 siRNA 在 I148M 纯合子 MAFLD 患者中的 1 期试验
PNPLA3 风险等位基因的纯合性与肝脏脂肪积累有关。在 1 期试验中，JNJ-75220795（一种针对肝细胞的 GalNAc 缀合的 PNPLA3 siRNA）可减少 PNPLA3 I148M 中的肝脏脂肪...
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