Literature DB >> 26046074

Gene therapy for GM1 gangliosidosis: challenges of translational medicine.

Carl Hayward1, Hitesh C Patel1, Sanjay G Manohar1, Alexander R Lyon1.   

Abstract

Entities:  

Year:  2015        PMID： 26046074      PMCID： PMC4437950          DOI： 10.3978/j.issn.2305-5839.2015.02.28

Source DB:  PubMed          Journal:  Ann Transl Med        ISSN： 2305-5839


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  18 in total

1.  Regulators split on gene therapy as patient shows signs of cancer.

Authors:  Erika Check
Journal:  Nature       Date:  2002-10-10       Impact factor: 49.962

2.  Regulatory evaluation of Glybera in Europe - two committees, one mission.

Authors:  Daniela Melchiorri; Luca Pani; Paolo Gasparini; Giulio Cossu; Janis Ancans; John Joseph Borg; Catherine Drai; Piotr Fiedor; Egbert Flory; Ian Hudson; Hubert G Leufkens; Jan Müller-Berghaus; Gopalan Narayanan; Brigitte Neugebauer; Juris Pokrotnieks; Jean-Louis Robert; Tomas Salmonson; Christian K Schneider
Journal:  Nat Rev Drug Discov       Date:  2013-08-19       Impact factor: 84.694

3.  Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.

Authors:  Stéphane Palfi; Jean Marc Gurruchaga; G Scott Ralph; Helene Lepetit; Sonia Lavisse; Philip C Buttery; Colin Watts; James Miskin; Michelle Kelleher; Sarah Deeley; Hirokazu Iwamuro; Jean Pascal Lefaucheur; Claire Thiriez; Gilles Fenelon; Cherry Lucas; Pierre Brugières; Inanna Gabriel; Kou Abhay; Xavier Drouot; Naoki Tani; Aurelie Kas; Bijan Ghaleh; Philippe Le Corvoisier; Patrice Dolphin; David P Breen; Sarah Mason; Natalie Valle Guzman; Nicholas D Mazarakis; Pippa A Radcliffe; Richard Harrop; Susan M Kingsman; Olivier Rascol; Stuart Naylor; Roger A Barker; Philippe Hantraye; Philippe Remy; Pierre Cesaro; Kyriacos A Mitrophanous
Journal:  Lancet       Date:  2014-01-10       Impact factor: 79.321

4.  Sustained normalization of neurological disease after intracranial gene therapy in a feline model.

Authors:  Victoria J McCurdy; Aime K Johnson; Heather L Gray-Edwards; Ashley N Randle; Brandon L Brunson; Nancy E Morrison; Nouha Salibi; Jacob A Johnson; Misako Hwang; Ronald J Beyers; Stanley G Leroy; Stacy Maitland; Thomas S Denney; Nancy R Cox; Henry J Baker; Miguel Sena-Esteves; Douglas R Martin
Journal:  Sci Transl Med       Date:  2014-04-09       Impact factor: 17.956

5.  Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.

Authors:  Kathrin Meyer; Laura Ferraiuolo; Leah Schmelzer; Lyndsey Braun; Vicki McGovern; Shibi Likhite; Olivia Michels; Alessandra Govoni; Julie Fitzgerald; Pablo Morales; Kevin D Foust; Jerry R Mendell; Arthur H M Burghes; Brian K Kaspar
Journal:  Mol Ther       Date:  2014-10-31       Impact factor: 11.454

6.  Chemical chaperone therapy: clinical effect in murine G(M1)-gangliosidosis.

Authors:  Yoshiyuki Suzuki; Satoshi Ichinomiya; Mieko Kurosawa; Masato Ohkubo; Hiroshi Watanabe; Hiroyuki Iwasaki; Junichiro Matsuda; Yoko Noguchi; Kazuhiro Takimoto; Masayuki Itoh; Miho Tabe; Masami Iida; Takatoshi Kubo; Seiichiro Ogawa; Eiji Nanba; Katsumi Higaki; Kousaku Ohno; Roscoe O Brady
Journal:  Ann Neurol       Date:  2007-12       Impact factor: 10.422

7.  Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.

Authors:  Junichiro Matsuda; Osamu Suzuki; Akihiro Oshima; Yoshie Yamamoto; Akira Noguchi; Kazuhiro Takimoto; Masayuki Itoh; Yuji Matsuzaki; Yosuke Yasuda; Seiichiro Ogawa; Yuko Sakata; Eiji Nanba; Katsumi Higaki; Yoshimi Ogawa; Lika Tominaga; Kousaku Ohno; Hiroyuki Iwasaki; Hiroshi Watanabe; Roscoe O Brady; Yoshiyuki Suzuki
Journal:  Proc Natl Acad Sci U S A       Date:  2003-12-15       Impact factor: 11.205

Review 8.  GM1 gangliosidosis: review of clinical, molecular, and therapeutic aspects.

Authors:  Nicola Brunetti-Pierri; Fernando Scaglia
Journal:  Mol Genet Metab       Date:  2008-06-03       Impact factor: 4.797

9.  Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer.

Authors:  N Takaura; T Yagi; M Maeda; E Nanba; A Oshima; Y Suzuki; T Yamano; A Tanaka
Journal:  Gene Ther       Date:  2003-08       Impact factor: 5.250

10.  Analysis of transduction efficiency, tropism and axonal transport of AAV serotypes 1, 2, 5, 6, 8 and 9 in the mouse brain.

Authors:  Dominik F Aschauer; Sebastian Kreuz; Simon Rumpel
Journal:  PLoS One       Date:  2013-09-27       Impact factor: 3.240
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  2 in total

1.  Intracellular Delivery of β-Galactosidase Enzyme Using Arginase-Responsive Dextran Sulfate/Poly-l-arginine Capsule for Lysosomal Storage Disorder.

Authors:  Meenakshi Gupta; Himanshu Pandey; Sri Sivakumar
Journal:  ACS Omega       Date:  2017-12-15

2.  Axonopathy and Reduction of Membrane Resistance: Key Features in a New Murine Model of Human GM1-Gangliosidosis.

Authors:  Deborah Eikelberg; Annika Lehmbecker; Graham Brogden; Witchaya Tongtako; Kerstin Hahn; Andre Habierski; Julia B Hennermann; Hassan Y Naim; Felix Felmy; Wolfgang Baumgärtner; Ingo Gerhauser
Journal:  J Clin Med       Date:  2020-04-02       Impact factor: 4.241
  2 in total

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