Leading RNA Interference Therapeutics Part 2: Silencing Delta-Aminolevulinic Acid Synthase 1, with a Focus on Givosiran
出版年份 2019 全文链接
标题
Leading RNA Interference Therapeutics Part 2: Silencing Delta-Aminolevulinic Acid Synthase 1, with a Focus on Givosiran
作者
关键词
-
出版物
Molecular Diagnosis & Therapy
Volume 24, Issue 1, Pages 61-68
出版商
Springer Science and Business Media LLC
发表日期
2019-12-03
DOI
10.1007/s40291-019-00438-6
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
- (2019) Eliane Sardh et al. NEW ENGLAND JOURNAL OF MEDICINE
- Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran
- (2019) Simoneide S. Titze-de-Almeida et al. Molecular Diagnosis & Therapy
- From a dominant to an oligogenic model of inheritance with environmental modifiers in acute intermittent porphyria
- (2018) Hugo Lenglet et al. HUMAN MOLECULAR GENETICS
- Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates
- (2018) Donald J. Foster et al. MOLECULAR THERAPY
- Journey of siRNA: Clinical Developments and Targeted Delivery
- (2018) Rahul R. Nikam et al. Nucleic Acid Therapeutics
- Porphyria
- (2017) D. Montgomery Bissell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates
- (2017) Jayaprakash K. Nair et al. NUCLEIC ACIDS RESEARCH
- Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs
- (2017) Xiulong Shen et al. NUCLEIC ACIDS RESEARCH
- The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market
- (2017) Ricardo Titze-de-Almeida et al. PHARMACEUTICAL RESEARCH
- Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics
- (2017) Yuanyu Huang Molecular Therapy-Nucleic Acids
- Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA
- (2016) Thazha P. Prakash et al. BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
- Update review of the acute porphyrias
- (2016) Penelope E. Stein et al. BRITISH JOURNAL OF HAEMATOLOGY
- Porphyria and Vampirism—A Myth, Sensationalized
- (2016) Eric Laurent Maranda et al. JAMA Dermatology
- siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes
- (2015) Shigeo Matsuda et al. ACS Chemical Biology
- Discovery of the magnetic behavior of hemoglobin: A beginning of bioinorganic chemistry
- (2015) Kara L. Bren et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification
- (2015) Amy Chan et al. Molecular Therapy-Nucleic Acids
- Liver transplantation in the management of porphyria
- (2014) Ashwani K. Singal et al. HEPATOLOGY
- Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
- (2014) Jayaprakash K. Nair et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice
- (2014) M. Yasuda et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- The incidence of inherited porphyrias in Europe
- (2012) George Elder et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Porphyrins: One Ring in the Colors of Life
- (2011) Franck Dayan et al. AMERICAN SCIENTIST
- Synthesis and characterization of 2′-modified-4′-thioRNA: a comprehensive comparison of nuclease stability
- (2009) Mayumi Takahashi et al. NUCLEIC ACIDS RESEARCH
- Trivalent, Gal/GalNAc-containing ligands designed for the asialoglycoprotein receptor
- (2008) Oleg Khorev et al. BIOORGANIC & MEDICINAL CHEMISTRY
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started