Literature DB >> 34433081

In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis.

Ana Rita Batista1, Terence R Flotte2.   

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Year:  2021        PMID: 34433081      PMCID: PMC8417911          DOI: 10.1016/j.ymthe.2021.08.014

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   12.910


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  10 in total

1.  Transthyretin: roles in the nervous system beyond thyroxine and retinol transport.

Authors:  Sandra Marisa Oliveira; Isabel Cardoso; Maria João Saraiva
Journal:  Expert Rev Endocrinol Metab       Date:  2012-03

2.  Tetramer dissociation and monomer partial unfolding precedes protofibril formation in amyloidogenic transthyretin variants.

Authors:  A Quintas; D C Vaz; I Cardoso; M J Saraiva; R M Brito
Journal:  J Biol Chem       Date:  2001-04-16       Impact factor: 5.157

Review 3.  The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug.

Authors:  Steven M Johnson; Stephen Connelly; Colleen Fearns; Evan T Powers; Jeffery W Kelly
Journal:  J Mol Biol       Date:  2012-01-05       Impact factor: 5.469

Review 4.  Emerging therapies in transthyretin amyloidosis - a new wave of hope after years of stagnancy?

Authors:  Maximilian L Müller; Javed Butler; Bettina Heidecker
Journal:  Eur J Heart Fail       Date:  2020-01-07       Impact factor: 15.534

5.  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

6.  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

7.  Safety and efficacy of RNAi therapy for transthyretin amyloidosis.

Authors:  Teresa Coelho; David Adams; Ana Silva; Pierre Lozeron; Philip N Hawkins; Timothy Mant; Javier Perez; Joseph Chiesa; Steve Warrington; Elizabeth Tranter; Malathy Munisamy; Rick Falzone; Jamie Harrop; Jeffrey Cehelsky; Brian R Bettencourt; Mary Geissler; James S Butler; Alfica Sehgal; Rachel E Meyers; Qingmin Chen; Todd Borland; Renta M Hutabarat; Valerie A Clausen; Rene Alvarez; Kevin Fitzgerald; Christina Gamba-Vitalo; Saraswathy V Nochur; Akshay K Vaishnaw; Dinah W Y Sah; Jared A Gollob; Ole B Suhr
Journal:  N Engl J Med       Date:  2013-08-29       Impact factor: 91.245

8.  Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

Authors:  Florie Borel; Qiushi Tang; Gwladys Gernoux; Cynthia Greer; Ziqiong Wang; Adi Barzel; Mark A Kay; Leonard D Shultz; Dale L Greiner; Terence R Flotte; Michael A Brehm; Christian Mueller
Journal:  Mol Ther       Date:  2017-09-25       Impact factor: 11.454

9.  Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy.

Authors:  Mathew S Maurer; Jeffrey H Schwartz; Balarama Gundapaneni; Perry M Elliott; Giampaolo Merlini; Marcia Waddington-Cruz; Arnt V Kristen; Martha Grogan; Ronald Witteles; Thibaud Damy; Brian M Drachman; Sanjiv J Shah; Mazen Hanna; Daniel P Judge; Alexandra I Barsdorf; Peter Huber; Terrell A Patterson; Steven Riley; Jennifer Schumacher; Michelle Stewart; Marla B Sultan; Claudio Rapezzi
Journal:  N Engl J Med       Date:  2018-08-27       Impact factor: 91.245

10.  CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

Authors:  Julian D Gillmore; Ed Gane; Jorg Taubel; Justin Kao; Marianna Fontana; Michael L Maitland; Jessica Seitzer; Daniel O'Connell; Kathryn R Walsh; Kristy Wood; Jonathan Phillips; Yuanxin Xu; Adam Amaral; Adam P Boyd; Jeffrey E Cehelsky; Mark D McKee; Andrew Schiermeier; Olivier Harari; Andrew Murphy; Christos A Kyratsous; Brian Zambrowicz; Randy Soltys; David E Gutstein; John Leonard; Laura Sepp-Lorenzino; David Lebwohl
Journal:  N Engl J Med       Date:  2021-06-26       Impact factor: 91.245

  10 in total

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