Literature DB >> 23998746

Prospects for stem cell therapy in neuronal ceroid lipofuscinosis.

Michael C Kruer, David A Pearce, Paul J Orchard, Robert D Steiner.   

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Year:  2013        PMID： 23998746      PMCID： PMC4421893          DOI： 10.2217/rme.13.46

Source DB:  PubMed          Journal:  Regen Med        ISSN： 1746-0751            Impact factor:   3.806


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  19 in total

1.  Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis.

Authors:  Shannon L Macauley; Marie S Roberts; Andrew M Wong; Francesca McSloy; Adarsh S Reddy; Jonathan D Cooper; Mark S Sands
Journal:  Ann Neurol       Date:  2012-02-24       Impact factor: 10.422

2.  Neural stem cell engraftment and myelination in the human brain.

Authors:  Nalin Gupta; Roland G Henry; Jonathan Strober; Sang-Mo Kang; Daniel A Lim; Monica Bucci; Eduardo Caverzasi; Laura Gaetano; Maria Luisa Mandelli; Tamara Ryan; Rachel Perry; Jody Farrell; Rita J Jeremy; Mary Ulman; Stephen L Huhn; A James Barkovich; David H Rowitch
Journal:  Sci Transl Med       Date:  2012-10-10       Impact factor: 17.956

3.  Confinement to organelle-associated inclusion structures mediates asymmetric inheritance of aggregated protein in budding yeast.

Authors:  Rachel Spokoini; Ofer Moldavski; Yaakov Nahmias; Jeremy L England; Maya Schuldiner; Daniel Kaganovich
Journal:  Cell Rep       Date:  2012-09-27       Impact factor: 9.423

4.  The blood-brain barrier is disrupted in a mouse model of infantile neuronal ceroid lipofuscinosis: amelioration by resveratrol.

Authors:  Arjun Saha; Chinmoy Sarkar; Satya P Singh; Zhongjian Zhang; Jeeva Munasinghe; Shiyong Peng; Goutam Chandra; Eryan Kong; Anil B Mukherjee
Journal:  Hum Mol Genet       Date:  2012-02-13       Impact factor: 6.150

5.  Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report.

Authors:  Weston P Miller; Steven M Rothman; David Nascene; Teresa Kivisto; Todd E DeFor; Richard S Ziegler; Julie Eisengart; Kara Leiser; Gerald Raymond; Troy C Lund; Jakub Tolar; Paul J Orchard
Journal:  Blood       Date:  2011-05-17       Impact factor: 22.113

6.  Immunosuppression alters disease severity in juvenile Batten disease mice.

Authors:  Sabrina S Seehafer; Denia Ramirez-Montealegre; Andrew Ms Wong; Chun-Hung Chan; Julian Castaneda; Michael Horak; Sarah M Ahmadi; Ming J Lim; Jonathan D Cooper; David A Pearce
Journal:  J Neuroimmunol       Date:  2011-01       Impact factor: 3.478

7.  An autoantibody inhibitory to glutamic acid decarboxylase in the neurodegenerative disorder Batten disease.

Authors:  Subrata Chattopadhyay; Masumi Ito; Jonathan D Cooper; Andrew I Brooks; Timothy M Curran; James M Powers; David A Pearce
Journal:  Hum Mol Genet       Date:  2002-06-01       Impact factor: 6.150

8.  Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.

Authors:  Nathalie Cartier; Salima Hacein-Bey-Abina; Cynthia C Bartholomae; Gabor Veres; Manfred Schmidt; Ina Kutschera; Michel Vidaud; Ulrich Abel; Liliane Dal-Cortivo; Laure Caccavelli; Nizar Mahlaoui; Véronique Kiermer; Denice Mittelstaedt; Céline Bellesme; Najiba Lahlou; François Lefrère; Stéphane Blanche; Muriel Audit; Emmanuel Payen; Philippe Leboulch; Bruno l'Homme; Pierre Bougnères; Christof Von Kalle; Alain Fischer; Marina Cavazzana-Calvo; Patrick Aubourg
Journal:  Science       Date:  2009-11-06       Impact factor: 47.728

9.  Large-scale phenotyping of an accurate genetic mouse model of JNCL identifies novel early pathology outside the central nervous system.

Authors:  John F Staropoli; Larissa Haliw; Sunita Biswas; Lillian Garrett; Sabine M Hölter; Lore Becker; Sergej Skosyrski; Patricia Da Silva-Buttkus; Julia Calzada-Wack; Frauke Neff; Birgit Rathkolb; Jan Rozman; Anja Schrewe; Thure Adler; Oliver Puk; Minxuan Sun; Jack Favor; Ildikó Racz; Raffi Bekeredjian; Dirk H Busch; Jochen Graw; Martin Klingenspor; Thomas Klopstock; Eckhard Wolf; Wolfgang Wurst; Andreas Zimmer; Edith Lopez; Hayat Harati; Eric Hill; Daniela S Krause; Jolene Guide; Ella Dragileva; Evan Gale; Vanessa C Wheeler; Rose-Mary Boustany; Diane E Brown; Sylvie Breton; Klaus Ruether; Valérie Gailus-Durner; Helmut Fuchs; Martin Hrabě de Angelis; Susan L Cotman
Journal:  PLoS One       Date:  2012-06-06       Impact factor: 3.240

10.  Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease.

Authors:  Carmine Spampanato; Erin Feeney; Lishu Li; Monica Cardone; Jeong-A Lim; Fabio Annunziata; Hossein Zare; Roman Polishchuk; Rosa Puertollano; Giancarlo Parenti; Andrea Ballabio; Nina Raben
Journal:  EMBO Mol Med       Date:  2013-04-18       Impact factor: 12.137
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  3 in total

Review 1.  Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy.

Authors:  Nicola Specchio; Alessandro Ferretti; Marina Trivisano; Nicola Pietrafusa; Chiara Pepi; Costanza Calabrese; Susanna Livadiotti; Alessandra Simonetti; Paolo Rossi; Paolo Curatolo; Federico Vigevano
Journal:  Drugs       Date:  2021-01       Impact factor: 9.546

2.  Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.

Authors:  Jonathan B Rosenberg; Alvin Chen; Stephen M Kaminsky; Ronald G Crystal; Dolan Sondhi
Journal:  Expert Opin Orphan Drugs       Date:  2019-11-27       Impact factor: 0.694

Review 3.  Neurophysiological Findings in Neuronal Ceroid Lipofuscinoses.

Authors:  Marina Trivisano; Alessandro Ferretti; Costanza Calabrese; Nicola Pietrafusa; Ludovica Piscitello; Giusy Carfi' Pavia; Federico Vigevano; Nicola Specchio
Journal:  Front Neurol       Date:  2022-02-25       Impact factor: 4.003
  3 in total

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