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Phase 1 Trials of PNPLA3 siRNA in I148M Homozygous Patients with MAFLD

by Pei, Tao , Ouchi, Shohei , Salazar, Hernan , Erion, Mark , Ayyar, Vivaswath S. , Rady, Brian , Koshkina, Anastasiya , Hamilton, Holly , Ding, Zhi-Ming , Shapiro, Craig , Jeon, Jae Yoon , Gargano, Cynthia , Afrazi, Maria , DiProspero, Nicholas , Yoshihara, Tatsuya , Pocai, Alessandro , Nicholas, Anthony , Inoue, Satoshi , Tamamura, Ryo , Makimura, Hideo , Saito, Yuki , Hegge, Julia , Nakano, Masayoshi , Matsuoka, Osamu , Fabbrini, Elisa , Yamashita, Aimi , Wendel, Susan , Yonemura, Takuma

in Acyltransferases - antagonists & inhibitors / Acyltransferases - genetics / Adult / Aged / Clinical trials / Drug dosages / Fatty liver / Female / Gastroenterology / Gastroenterology General / Genetics / Genetics General / Hispanic Americans / Homozygote / Humans / Liver / Liver - drug effects / Liver - pathology / Liver Disease / Liver diseases / Male / Metabolism / Middle Aged / Mutation / Non-alcoholic Fatty Liver Disease - drug therapy / Non-alcoholic Fatty Liver Disease - genetics / Non-alcoholic Fatty Liver Disease - metabolism / Non-alcoholic Fatty Liver Disease - pathology / Phospholipases A2, Calcium-Independent - antagonists & inhibitors / Phospholipases A2, Calcium-Independent - genetics / Polymorphism, Single Nucleotide / Precision Medicine - methods / Proof of Concept Study / RNA Therapeutics / RNA, Small Interfering - administration & dosage / RNA, Small Interfering - adverse effects / RNA, Small Interfering - pharmacokinetics / siRNA / Young Adult

2024

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Phase 1 Trials of PNPLA3 siRNA in I148M Homozygous Patients with MAFLD

2024

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Phase 1 Trials of PNPLA3 siRNA in I148M Homozygous Patients with MAFLD

2024

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Journal Article

Phase 1 Trials of PNPLA3 siRNA in I148M Homozygous Patients with MAFLD

Pei, Tao,

Ouchi, Shohei,

Salazar, Hernan,

Erion, Mark,

Ayyar, Vivaswath S.,

Rady, Brian,

Koshkina, Anastasiya,

Hamilton, Holly,

Ding, Zhi-Ming,

Shapiro, Craig,

Jeon, Jae Yoon,

Gargano, Cynthia,

Afrazi, Maria,

DiProspero, Nicholas,

Yoshihara, Tatsuya,

Pocai, Alessandro,

Nicholas, Anthony,

Inoue, Satoshi,

Tamamura, Ryo,

Makimura, Hideo,

Saito, Yuki,

Hegge, Julia,

Nakano, Masayoshi,

Matsuoka, Osamu,

Fabbrini, Elisa,

Yamashita, Aimi,

Wendel, Susan,

Yonemura, Takuma

2024

Overview

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 homozygous patients with metabolic dysfunction–associated fatty liver disease.

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Publisher

Massachusetts Medical Society

Subject

Acyltransferases - antagonists & inhibitors

/ Acyltransferases - genetics

/ Adult

/ Aged

/ Clinical trials

/ Drug dosages

/ Fatty liver