Literature DB >> 17049436

Neuronal ceroid lipofuscinoses therapeutic strategies: past, present and future.

Judith A Hobert1, Glyn Dawson.   

Abstract

Historically, many different therapies have been assessed for their ability to alter disease progression of the Neuronal Ceroid Lipofuscinoses (NCLs). While some treatments have lead to minor improvements, none have been able to arrest disease progression or improve the quality or duration of life. Presently, many new therapeutic strategies, such as chaperone therapy, enzyme replacement therapy, gene therapy, and stem cell therapy, are being investigated for their ability to alter the disease course of the NCLs. This review summarizes previous studied therapies, discusses those currently being evaluated and examines possibilities for future therapies for the treatment of patients with NCL.

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Year:  2006        PMID： 17049436     DOI： 10.1016/j.bbadis.2006.08.004

Source DB:  PubMed          Journal:  Biochim Biophys Acta        ISSN： 0006-3002


  18 in total

Review 1.  Research challenges in central nervous system manifestations of inborn errors of metabolism.

Authors:  P I Dickson; A R Pariser; S C Groft; R W Ishihara; D E McNeil; D Tagle; D J Griebel; S G Kaler; J W Mink; E G Shapiro; K J Bjoraker; L Krivitzky; J M Provenzale; A Gropman; P Orchard; G Raymond; B H Cohen; R D Steiner; S F Goldkind; R M Nelson; E Kakkis; M C Patterson
Journal:  Mol Genet Metab       Date:  2010-12-02       Impact factor: 4.797

Review 2.  Quantum dots and potential therapy for Krabbe's disease.

Authors:  Glyn Dawson
Journal:  J Neurosci Res       Date:  2016-11       Impact factor: 4.164

3.  Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system.

Authors:  Hongwei Zhang; Bin Yang; Xin Mu; Seemin Seher Ahmed; Qin Su; Ran He; Hongyan Wang; Christian Mueller; Miguel Sena-Esteves; Robert Brown; Zuoshang Xu; Guangping Gao
Journal:  Mol Ther       Date:  2011-05-24       Impact factor: 11.454

4.  Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients.

Authors:  Chinmoy Sarkar; Zhongjian Zhang; Anil B Mukherjee
Journal:  Mol Genet Metab       Date:  2011-06-13       Impact factor: 4.797

Review 5.  Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.

Authors:  R Kohan; I A Cismondi; A M Oller-Ramirez; N Guelbert; Tapia V Anzolini; G Alonso; S E Mole; Dodelson R de Kremer; Noher I de Halac
Journal:  Curr Pharm Biotechnol       Date:  2011-06       Impact factor: 2.837

Review 6.  Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases.

Authors:  Lamya S Shihabuddin; Seng H Cheng
Journal:  Neurotherapeutics       Date:  2011-10       Impact factor: 7.620

7.  Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System.

Authors:  Dominic J Gessler; Phillip W L Tai; Jia Li; Guangping Gao
Journal:  Methods Mol Biol       Date:  2019

8.  Substrate Reduction Therapy in Four Patients with Milder CLN1 Mutations and Juvenile-Onset Batten Disease Using Cysteamine Bitartrate.

Authors:  M Gavin; G Y Wen; J Messing; S Adelman; A Logush; E C Jenkins; W T Brown; M Velinov
Journal:  JIMD Rep       Date:  2013-04-16

9.  Palmitoyl protein thioesterase-1 deficiency impairs synaptic vesicle recycling at nerve terminals, contributing to neuropathology in humans and mice.

Authors:  Sung-Jo Kim; Zhongjian Zhang; Chinmoy Sarkar; Pei-Chih Tsai; Yi-Ching Lee; Louis Dye; Anil B Mukherjee
Journal:  J Clin Invest       Date:  2008-09       Impact factor: 14.808

Review 10.  Progress in the Development of Small Molecule Therapeutics for the Treatment of Neuronal Ceroid Lipofuscinoses (NCLs).

Authors:  Nihar Kinarivala; Paul C Trippier
Journal:  J Med Chem       Date:  2015-11-24       Impact factor: 7.446
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