Literature DB >> 31037650

Nucleic Acid Therapeutics for Neurological Diseases.

Jonathan K Watts1, Robert H Brown2,3, Anastasia Khvorova2.   

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Year:  2019        PMID： 31037650      PMCID： PMC6554381          DOI： 10.1007/s13311-019-00736-1

Source DB:  PubMed          Journal:  Neurotherapeutics        ISSN： 1878-7479            Impact factor:   7.620


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  14 in total

Review 1.  Informational drugs: a new concept in pharmacology.

Authors:  J S Cohen
Journal:  Antisense Res Dev       Date:  1991

2.  FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides.

Authors:  Annemieke Aartsma-Rus
Journal:  Nucleic Acid Ther       Date:  2017-02-21       Impact factor: 5.486

Review 3.  Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy.

Authors:  David R Corey
Journal:  Nat Neurosci       Date:  2017-02-13       Impact factor: 24.884

Review 4.  The chemical evolution of oligonucleotide therapies of clinical utility.

Authors:  Anastasia Khvorova; Jonathan K Watts
Journal:  Nat Biotechnol       Date:  2017-02-27       Impact factor: 54.908

Review 5.  Nucleic Acid Therapies for Ischemic Stroke.

Authors:  Nils Henninger; Yunis Mayasi
Journal:  Neurotherapeutics       Date:  2019-04       Impact factor: 7.620

6.  Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.

Authors:  David Adams; Alejandra Gonzalez-Duarte; William D O'Riordan; Chih-Chao Yang; Mitsuharu Ueda; Arnt V Kristen; Ivailo Tournev; Hartmut H Schmidt; Teresa Coelho; John L Berk; Kon-Ping Lin; Giuseppe Vita; Shahram Attarian; Violaine Planté-Bordeneuve; Michelle M Mezei; Josep M Campistol; Juan Buades; Thomas H Brannagan; Byoung J Kim; Jeeyoung Oh; Yesim Parman; Yoshiki Sekijima; Philip N Hawkins; Scott D Solomon; Michael Polydefkis; Peter J Dyck; Pritesh J Gandhi; Sunita Goyal; Jihong Chen; Andrew L Strahs; Saraswathy V Nochur; Marianne T Sweetser; Pushkal P Garg; Akshay K Vaishnaw; Jared A Gollob; Ole B Suhr
Journal:  N Engl J Med       Date:  2018-07-05       Impact factor: 91.245

7.  Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.

Authors:  Merrill D Benson; Márcia Waddington-Cruz; John L Berk; Michael Polydefkis; Peter J Dyck; Annabel K Wang; Violaine Planté-Bordeneuve; Fabio A Barroso; Giampaolo Merlini; Laura Obici; Morton Scheinberg; Thomas H Brannagan; William J Litchy; Carol Whelan; Brian M Drachman; David Adams; Stephen B Heitner; Isabel Conceição; Hartmut H Schmidt; Giuseppe Vita; Josep M Campistol; Josep Gamez; Peter D Gorevic; Edward Gane; Amil M Shah; Scott D Solomon; Brett P Monia; Steven G Hughes; T Jesse Kwoh; Bradley W McEvoy; Shiangtung W Jung; Brenda F Baker; Elizabeth J Ackermann; Morie A Gertz; Teresa Coelho
Journal:  N Engl J Med       Date:  2018-07-05       Impact factor: 91.245

8.  Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial.

Authors:  Frederick J Raal; Raul D Santos; Dirk J Blom; A David Marais; Min-Ji Charng; William C Cromwell; Robin H Lachmann; Daniel Gaudet; Ju L Tan; Scott Chasan-Taber; Diane L Tribble; Joann D Flaim; Stanley T Crooke
Journal:  Lancet       Date:  2010-03-20       Impact factor: 79.321

9.  Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.

Authors:  Claudia A Chiriboga; Kathryn J Swoboda; Basil T Darras; Susan T Iannaccone; Jacqueline Montes; Darryl C De Vivo; Daniel A Norris; C Frank Bennett; Kathie M Bishop
Journal:  Neurology       Date:  2016-02-10       Impact factor: 9.910

Review 10.  Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias.

Authors:  Ronald A M Buijsen; Lodewijk J A Toonen; Sarah L Gardiner; Willeke M C van Roon-Mom
Journal:  Neurotherapeutics       Date:  2019-04       Impact factor: 7.620
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  7 in total

Review 1.  Beyond GalNAc! Drug delivery systems comprising complex oligosaccharides for targeted use of nucleic acid therapeutics.

Authors:  Joseph O'Sullivan; Jose Muñoz-Muñoz; Graeme Turnbull; Neil Sim; Stuart Penny; Sterghios Moschos
Journal:  RSC Adv       Date:  2022-07-14       Impact factor: 4.036

Review 2.  Cerebral Organoids and Antisense Oligonucleotide Therapeutics: Challenges and Opportunities.

Authors:  Jenny Lange; Haiyan Zhou; Amy McTague
Journal:  Front Mol Neurosci       Date:  2022-06-27       Impact factor: 6.261

3.  Length Polymorphism and Methylation Status of UPS29 Minisatellite of the ACAP3 Gene as Molecular Biomarker of Epilepsy. Sex Differences in Seizure Types and Symptoms.

Authors:  Irina O Suchkova; Elena V Borisova; Eugene L Patkin
Journal:  Int J Mol Sci       Date:  2020-12-02       Impact factor: 5.923

Review 4.  Intranasal delivery: An attractive route for the administration of nucleic acid based therapeutics for CNS disorders.

Authors:  Pranav Shah; Manisha Lalan; Kalyani Barve
Journal:  Front Pharmacol       Date:  2022-08-30       Impact factor: 5.988

Review 5.  Nucleic Acid Nanoparticles at a Crossroads of Vaccines and Immunotherapies.

Authors:  Marina A Dobrovolskaia
Journal:  Molecules       Date:  2019-12-17       Impact factor: 4.411

Review 6.  Small Molecule Drugs Targeting Non-Coding RNAs as Treatments for Alzheimer's Disease and Related Dementias.

Authors:  Lien D Nguyen; Rachel K Chau; Anna M Krichevsky
Journal:  Genes (Basel)       Date:  2021-12-17       Impact factor: 4.096

Review 7.  Advances in the Design of (Nano)Formulations for Delivery of Antisense Oligonucleotides and Small Interfering RNA: Focus on the Central Nervous System.

Authors:  Monique C P Mendonça; Ayse Kont; Maria Rodriguez Aburto; John F Cryan; Caitriona M O'Driscoll
Journal:  Mol Pharm       Date:  2021-03-18       Impact factor: 4.939
  7 in total

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