Literature DB >> 25985262

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency.

Azita J Mahiny1, Alexander Dewerth1, Lauren E Mays1, Mohammed Alkhaled1, Benedikt Mothes2, Emad Malaeksefat3, Brigitta Loretz3, Jennifer Rottenberger1, Darina M Brosch1, Philipp Reautschnig1, Pacharapan Surapolchai1, Franziska Zeyer1, Andrea Schams4, Melanie Carevic1, Martina Bakele1, Matthias Griese4, Matthias Schwab5, Bernd Nürnberg6, Sandra Beer-Hammer6, Rupert Handgretinger1, Dominik Hartl1, Claus-Michael Lehr7, Michael S D Kormann1.   

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Year:  2015        PMID: 25985262     DOI: 10.1038/nbt.3241

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


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  17 in total

1.  Preparation and characterization of cationic PLGA nanospheres as DNA carriers.

Authors:  M N V Ravi Kumar; U Bakowsky; C M Lehr
Journal:  Biomaterials       Date:  2004-05       Impact factor: 12.479

2.  Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability.

Authors:  Katalin Karikó; Hiromi Muramatsu; Frank A Welsh; János Ludwig; Hiroki Kato; Shizuo Akira; Drew Weissman
Journal:  Mol Ther       Date:  2008-09-16       Impact factor: 11.454

3.  Characterization of airway and vascular responses in murine lungs.

Authors:  H D Held; C Martin; S Uhlig
Journal:  Br J Pharmacol       Date:  1999-03       Impact factor: 8.739

4.  Cationic poly(lactide-co-glycolide) nanoparticles as efficient in vivo gene transfection agents.

Authors:  M N V Ravi Kumar; S S Mohapatra; X Kong; P K Jena; U Bakowsky; C M Lehr
Journal:  J Nanosci Nanotechnol       Date:  2004-11

5.  Highly efficient endogenous human gene correction using designed zinc-finger nucleases.

Authors:  Fyodor D Urnov; Jeffrey C Miller; Ya-Li Lee; Christian M Beausejour; Jeremy M Rock; Sheldon Augustus; Andrew C Jamieson; Matthew H Porteus; Philip D Gregory; Michael C Holmes
Journal:  Nature       Date:  2005-04-03       Impact factor: 49.962

6.  Expression of therapeutic proteins after delivery of chemically modified mRNA in mice.

Authors:  Michael S D Kormann; Günther Hasenpusch; Manish K Aneja; Gabriela Nica; Andreas W Flemmer; Susanne Herber-Jonat; Marceline Huppmann; Lauren E Mays; Marta Illenyi; Andrea Schams; Matthias Griese; Iris Bittmann; Rupert Handgretinger; Dominik Hartl; Joseph Rosenecker; Carsten Rudolph
Journal:  Nat Biotechnol       Date:  2011-01-09       Impact factor: 54.908

7.  Systemic delivery of modified mRNA encoding herpes simplex virus 1 thymidine kinase for targeted cancer gene therapy.

Authors:  Yuhua Wang; Hsing-Hao Su; Yang Yang; Yunxia Hu; Lu Zhang; Pilar Blancafort; Leaf Huang
Journal:  Mol Ther       Date:  2012-12-11       Impact factor: 11.454

8.  Modified Foxp3 mRNA protects against asthma through an IL-10-dependent mechanism.

Authors:  Lauren E Mays; Susanne Ammon-Treiber; Benedikt Mothes; Mohammed Alkhaled; Jennifer Rottenberger; Eva Sophie Müller-Hermelink; Melanie Grimm; Markus Mezger; Sandra Beer-Hammer; Esther von Stebut; Nikolaus Rieber; Bernd Nürnberg; Matthias Schwab; Rupert Handgretinger; Marco Idzko; Dominik Hartl; Michael S D Kormann
Journal:  J Clin Invest       Date:  2013-02-08       Impact factor: 14.808

9.  Efficient control of gene expression by single step integration of the tetracycline system in transgenic mice.

Authors:  N Schultze; Y Burki; Y Lang; U Certa; H Bluethmann
Journal:  Nat Biotechnol       Date:  1996-04       Impact factor: 54.908

10.  Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases.

Authors:  Yannick Doyon; Jasmine M McCammon; Jeffrey C Miller; Farhoud Faraji; Catherine Ngo; George E Katibah; Rainier Amora; Toby D Hocking; Lei Zhang; Edward J Rebar; Philip D Gregory; Fyodor D Urnov; Sharon L Amacher
Journal:  Nat Biotechnol       Date:  2008-05-25       Impact factor: 54.908

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  45 in total

Review 1.  Salient Features of Endonuclease Platforms for Therapeutic Genome Editing.

Authors:  Michael T Certo; Richard A Morgan
Journal:  Mol Ther       Date:  2016-01-22       Impact factor: 11.454

Review 2.  Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery.

Authors:  Piotr S Kowalski; Arnab Rudra; Lei Miao; Daniel G Anderson
Journal:  Mol Ther       Date:  2019-02-19       Impact factor: 11.454

Review 3.  Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9.

Authors:  Hong-Xia Zhang; Ying Zhang; Hao Yin
Journal:  Mol Ther       Date:  2019-01-25       Impact factor: 11.454

Review 4.  Delivery technologies for genome editing.

Authors:  Hao Yin; Kevin J Kauffman; Daniel G Anderson
Journal:  Nat Rev Drug Discov       Date:  2017-03-24       Impact factor: 84.694

Review 5.  Diverse Applications of Nanomedicine.

Authors:  Beatriz Pelaz; Christoph Alexiou; Ramon A Alvarez-Puebla; Frauke Alves; Anne M Andrews; Sumaira Ashraf; Lajos P Balogh; Laura Ballerini; Alessandra Bestetti; Cornelia Brendel; Susanna Bosi; Monica Carril; Warren C W Chan; Chunying Chen; Xiaodong Chen; Xiaoyuan Chen; Zhen Cheng; Daxiang Cui; Jianzhong Du; Christian Dullin; Alberto Escudero; Neus Feliu; Mingyuan Gao; Michael George; Yury Gogotsi; Arnold Grünweller; Zhongwei Gu; Naomi J Halas; Norbert Hampp; Roland K Hartmann; Mark C Hersam; Patrick Hunziker; Ji Jian; Xingyu Jiang; Philipp Jungebluth; Pranav Kadhiresan; Kazunori Kataoka; Ali Khademhosseini; Jindřich Kopeček; Nicholas A Kotov; Harald F Krug; Dong Soo Lee; Claus-Michael Lehr; Kam W Leong; Xing-Jie Liang; Mei Ling Lim; Luis M Liz-Marzán; Xiaowei Ma; Paolo Macchiarini; Huan Meng; Helmuth Möhwald; Paul Mulvaney; Andre E Nel; Shuming Nie; Peter Nordlander; Teruo Okano; Jose Oliveira; Tai Hyun Park; Reginald M Penner; Maurizio Prato; Victor Puntes; Vincent M Rotello; Amila Samarakoon; Raymond E Schaak; Youqing Shen; Sebastian Sjöqvist; Andre G Skirtach; Mahmoud G Soliman; Molly M Stevens; Hsing-Wen Sung; Ben Zhong Tang; Rainer Tietze; Buddhisha N Udugama; J Scott VanEpps; Tanja Weil; Paul S Weiss; Itamar Willner; Yuzhou Wu; Lily Yang; Zhao Yue; Qian Zhang; Qiang Zhang; Xian-En Zhang; Yuliang Zhao; Xin Zhou; Wolfgang J Parak
Journal:  ACS Nano       Date:  2017-03-14       Impact factor: 15.881

Review 6.  Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems.

Authors:  S Guan; J Rosenecker
Journal:  Gene Ther       Date:  2017-01-17       Impact factor: 5.250

Review 7.  Non-viral delivery of genome-editing nucleases for gene therapy.

Authors:  M Wang; Z A Glass; Q Xu
Journal:  Gene Ther       Date:  2016-10-31       Impact factor: 5.250

Review 8.  Genome-Editing Technologies: Principles and Applications.

Authors:  Thomas Gaj; Shannon J Sirk; Sai-Lan Shui; Jia Liu
Journal:  Cold Spring Harb Perspect Biol       Date:  2016-12-01       Impact factor: 10.005

Review 9.  Impact of gene editing on the study of cystic fibrosis.

Authors:  Patrick T Harrison; David J Sanz; Jennifer A Hollywood
Journal:  Hum Genet       Date:  2016-06-21       Impact factor: 4.132

Review 10.  Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.

Authors:  Thomas Gaj; Benjamin E Epstein; David V Schaffer
Journal:  Mol Ther       Date:  2015-09-16       Impact factor: 11.454

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