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Literature DB >> 29121028

With Alnylam's amyloidosis success, RNAi approval hopes soar.

Cormac Sheridan.

Abstract

Entities: Disease

Mesh：

Substances：

Year: 2017 PMID： 29121028 DOI： 10.1038/nbt1117-995

Source DB: PubMed Journal: Nat Biotechnol ISSN： 1087-0156 Impact factor: 54.908

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7 in total

1. Worth the RISC?

Authors: Ken Garber
Journal: Nat Biotechnol Date: 2017-02-27 Impact factor: 54.908

2. Alnylam terminates revusiran program, stock plunges.

Authors: Ken Garber
Journal: Nat Biotechnol Date: 2016-12-07 Impact factor: 54.908

3. Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis.

Authors: M B Pepys; J Herbert; W L Hutchinson; G A Tennent; H J Lachmann; J R Gallimore; L B Lovat; T Bartfai; A Alanine; C Hertel; T Hoffmann; R Jakob-Roetne; R D Norcross; J A Kemp; K Yamamura; M Suzuki; G W Taylor; S Murray; D Thompson; A Purvis; S Kolstoe; S P Wood; P N Hawkins
Journal: Nature Date: 2002-05-16 Impact factor: 49.962

4. Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol.

Authors: Kausik K Ray; Ulf Landmesser; Lawrence A Leiter; David Kallend; Robert Dufour; Mahir Karakas; Tim Hall; Roland P T Troquay; Traci Turner; Frank L J Visseren; Peter Wijngaard; R Scott Wright; John J P Kastelein
Journal: N Engl J Med Date: 2017-03-17 Impact factor: 91.245

5. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.

Authors: John L Berk; Ole B Suhr; Laura Obici; Yoshiki Sekijima; Steven R Zeldenrust; Taro Yamashita; Michael A Heneghan; Peter D Gorevic; William J Litchy; Janice F Wiesman; Erik Nordh; Manuel Corato; Alessandro Lozza; Andrea Cortese; Jessica Robinson-Papp; Theodore Colton; Denis V Rybin; Alice B Bisbee; Yukio Ando; Shu-ichi Ikeda; David C Seldin; Giampaolo Merlini; Martha Skinner; Jeffery W Kelly; Peter J Dyck
Journal: JAMA Date: 2013-12-25 Impact factor: 56.272

6. Novel conformation-specific monoclonal antibodies against amyloidogenic forms of transthyretin.

Authors: Jeffrey N Higaki; Avi Chakrabartty; Natalie J Galant; Kevin C Hadley; Bradley Hammerson; Tarlochan Nijjar; Ronald Torres; Jose R Tapia; Joshua Salmans; Robin Barbour; Stephen J Tam; Ken Flanagan; Wagner Zago; Gene G Kinney
Journal: Amyloid Date: 2016-03-16 Impact factor: 7.141

7. Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy.

Authors: David Adams; Ole B Suhr; Peter J Dyck; William J Litchy; Raina G Leahy; Jihong Chen; Jared Gollob; Teresa Coelho
Journal: BMC Neurol Date: 2017-09-11 Impact factor: 2.474

7 in total

4 in total

With Alnylam's amyloidosis success, RNAi approval hopes soar.

1. Worth the RISC?

2. Alnylam terminates revusiran program, stock plunges.

3. Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis.

4. Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol.

5. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.

6. Novel conformation-specific monoclonal antibodies against amyloidogenic forms of transthyretin.

7. Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy.

1. Label-Free Electrophoretic Mobility Shift Assay (EMSA) for Measuring Dissociation Constants of Protein-RNA Complexes.

2. A Potent Branched-Tail Lipid Nanoparticle Enables Multiplexed mRNA Delivery and Gene Editing In Vivo.

Review 3. siRNA Specificity: RNAi Mechanisms and Strategies to Reduce Off-Target Effects.

Review 4. Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.