Literature DB >> 32909632

Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor?

Glenn F Pierce1, Radoslaw Kaczmarek2, Declan Noone3, Brian O'Mahony4, David Page5, Mark W Skinner6.   

Abstract

Entities:  

Year:  2020        PMID： 32909632     DOI： 10.1111/hae.14131

Source DB:  PubMed          Journal:  Haemophilia        ISSN： 1351-8216            Impact factor:   4.287


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  9 in total

1.  Making friends out of foes: novel therapeutic approaches for haemophilia.

Authors:  Catriona Mactier; Cedric Hermans
Journal:  Blood Transfus       Date:  2020-12-23       Impact factor: 3.443

2.  Eliminating Panglossian thinking in development of AAV therapeutics.

Authors:  Radoslaw Kaczmarek; Glenn F Pierce; Declan Noone; Brian O'Mahony; David Page; Mark W Skinner
Journal:  Mol Ther       Date:  2021-11-10       Impact factor: 11.454

3.  The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making.

Authors:  Michael Wang; Claude Negrier; Frank Driessler; Clifford Goodman; Mark W Skinner
Journal:  Patient Prefer Adherence       Date:  2022-06-09       Impact factor: 2.314

4.  Haemophilia care: the only constant is change.

Authors:  Benjamin J Samelson-Jones; Lindsey A George
Journal:  Br J Haematol       Date:  2021-08-02       Impact factor: 6.998

5.  Treatment-induced hemophilic thrombosis?

Authors:  Radoslaw Kaczmarek; Roland W Herzog
Journal:  Mol Ther       Date:  2022-01-20       Impact factor: 11.454

6.  Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.

Authors:  Eline van Overbeeke; Sissel Michelsen; Brett Hauber; Kathelijne Peerlinck; Cedric Hermans; Catherine Lambert; Michel Goldman; Steven Simoens; Isabelle Huys
Journal:  Haemophilia       Date:  2020-11-07       Impact factor: 4.287

7.  Patient selection for hemophilia gene therapy: Real-life data from a single center.

Authors:  Evelien Krumb; Catherine Lambert; Cedric Hermans
Journal:  Res Pract Thromb Haemost       Date:  2021-03-14

8.  Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette.

Authors:  Sandeep R P Kumar; Jun Xie; Shilang Hu; Jihye Ko; Qifeng Huang; Harrison C Brown; Alok Srivastava; David M Markusic; Christopher B Doering; H Trent Spencer; Arun Srivastava; Guangping Gao; Roland W Herzog
Journal:  Mol Ther Methods Clin Dev       Date:  2021-08-26       Impact factor: 6.698

9.  Gene therapy - are we ready now?

Authors:  Radoslaw Kaczmarek
Journal:  Haemophilia       Date:  2022-05       Impact factor: 4.263
  9 in total

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