Literature DB >> 30903542

Theranostics of Genetic Diseases.

Roberto Gambari1, Marina Kleanthous2.   

Abstract

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Year:  2019        PMID： 30903542     DOI： 10.1007/s40291-019-00395-0

Source DB:  PubMed          Journal:  Mol Diagn Ther        ISSN： 1177-1062            Impact factor:   4.074


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  23 in total

1.  MicroRNA-15a and -16-1 act via MYB to elevate fetal hemoglobin expression in human trisomy 13.

Authors:  Vijay G Sankaran; Tobias F Menne; Danilo Šćepanović; Jo-Anne Vergilio; Peng Ji; Jinkuk Kim; Prathapan Thiru; Stuart H Orkin; Eric S Lander; Harvey F Lodish
Journal:  Proc Natl Acad Sci U S A       Date:  2011-01-04       Impact factor: 11.205

Review 2.  Diagnosis and therapy with CRISPR advanced CRISPR based tools for point of care diagnostics and early therapies.

Authors:  Vanita Uppada; Mahesh Gokara; Girish Kumar Rasineni
Journal:  Gene       Date:  2018-02-27       Impact factor: 3.688

3.  Gene Therapy as a Curative Option for β-Thalassemia.

Authors:  Alessandra Biffi
Journal:  N Engl J Med       Date:  2018-04-19       Impact factor: 91.245

Review 4.  Innovative Therapies for Cystic Fibrosis: The Road from Treatment to Cure.

Authors:  Giulio Cabrini
Journal:  Mol Diagn Ther       Date:  2019-04       Impact factor: 4.074

Review 5.  MicroRNA therapeutics: towards a new era for the management of cancer and other diseases.

Authors:  Rajesha Rupaimoole; Frank J Slack
Journal:  Nat Rev Drug Discov       Date:  2017-02-17       Impact factor: 84.694

Review 6.  Gene editing for inflammatory disorders.

Authors:  David T Ewart; Erik J Peterson; Clifford J Steer
Journal:  Ann Rheum Dis       Date:  2018-08-04       Impact factor: 19.103

Review 7.  Gene Therapy and Genome Editing.

Authors:  Farid Boulad; Jorge Mansilla-Soto; Annalisa Cabriolu; Isabelle Rivière; Michel Sadelain
Journal:  Hematol Oncol Clin North Am       Date:  2018-01-09       Impact factor: 3.722

8.  Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing.

Authors:  Chengzu Long; Hui Li; Malte Tiburcy; Cristina Rodriguez-Caycedo; Viktoriia Kyrychenko; Huanyu Zhou; Yu Zhang; Yi-Li Min; John M Shelton; Pradeep P A Mammen; Norman Y Liaw; Wolfram-Hubertus Zimmermann; Rhonda Bassel-Duby; Jay W Schneider; Eric N Olson
Journal:  Sci Adv       Date:  2018-01-31       Impact factor: 14.136

Review 9.  Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine.

Authors:  Chiranjib Chakraborty; Ashish Ranjan Sharma; Garima Sharma; C George Priya Doss; Sang-Soo Lee
Journal:  Mol Ther Nucleic Acids       Date:  2017-06-12       Impact factor: 8.886

Review 10.  Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases.

Authors:  Panayiota Papasavva; Marina Kleanthous; Carsten W Lederer
Journal:  Mol Diagn Ther       Date:  2019-04       Impact factor: 4.074
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