Literature DB >> 11708986

Feasibility of gene therapy for late neuronal ceroid lipofuscinosis.

D Sondhi1, N R Hackett, R L Apblett, S M Kaminsky, R G Pergolizzi, R G Crystal.   

Abstract

Late infantile neuronal ceroid lipofuscinosis is a progressive childhood neurodegenerative disorder characterized by intracellular accumulation of autofluorescent material resembling lipofuscin in neuronal cells. This report summarizes the new therapies under consideration for late infantile neuronal ceroid lipofuscinosis, with a focus on strategies for in vivo gene therapy for the retinal and central nervous system manifestations of the disease.

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Year:  2001        PMID： 11708986     DOI： 10.1001/archneur.58.11.1793

Source DB:  PubMed          Journal:  Arch Neurol        ISSN： 0003-9942


  10 in total

1.  Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.

Authors:  Mark M Souweidane; Justin F Fraser; Lisa M Arkin; Dolan Sondhi; Neil R Hackett; Stephen M Kaminsky; Linda Heier; Barry E Kosofsky; Stefan Worgall; Ronald G Crystal; Michael G Kaplitt
Journal:  J Neurosurg Pediatr       Date:  2010-08       Impact factor: 2.375

2.  Upregulation of tripeptidyl-peptidase 1 by 3-hydroxy-(2,2)-dimethyl butyrate, a brain endogenous ligand of PPARα: Implications for late-infantile Batten disease therapy.

Authors:  Sudipta Chakrabarti; Sujyoti Chandra; Avik Roy; Sridevi Dasarathi; Madhuchhanda Kundu; Kalipada Pahan
Journal:  Neurobiol Dis       Date:  2019-03-28       Impact factor: 5.996

Review 3.  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

4.  Role of stem cells in treatment of neurological disorder.

Authors:  Ashfaq Ul Hassan; Ghulam Hassan; Zahida Rasool
Journal:  Int J Health Sci (Qassim)       Date:  2009-07

5.  Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging.

Authors:  J P Dyke; D Sondhi; H U Voss; D C Shungu; X Mao; K Yohay; S Worgall; N R Hackett; C Hollmann; M E Yeotsas; A L Jeong; B Van de Graaf; I Cao; S M Kaminsky; L A Heier; K D Rudser; M M Souweidane; M G Kaplitt; B Kosofsky; R G Crystal; D Ballon
Journal:  AJNR Am J Neuroradiol       Date:  2012-10-04       Impact factor: 3.825

6.  Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis.

Authors:  Dolan Sondhi; Daniel A Peterson; Andrew M Edelstein; Katrina del Fierro; Neil R Hackett; Ronald G Crystal
Journal:  Exp Neurol       Date:  2008-04-30       Impact factor: 5.330

7.  Assessing disease severity in late infantile neuronal ceroid lipofuscinosis using quantitative MR diffusion-weighted imaging.

Authors:  J P Dyke; H U Voss; D Sondhi; N R Hackett; S Worgall; L A Heier; B E Kosofsky; A M Uluğ; D C Shungu; X Mao; R G Crystal; D Ballon
Journal:  AJNR Am J Neuroradiol       Date:  2007-08       Impact factor: 3.825

8.  Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.

Authors:  Dolan Sondhi; Linda Johnson; Keith Purpura; Sebastien Monette; Mark M Souweidane; Michael G Kaplitt; Barry Kosofsky; Kaleb Yohay; Douglas Ballon; Jonathan Dyke; Stephen M Kaminksy; Neil R Hackett; Ronald G Crystal
Journal:  Hum Gene Ther Methods       Date:  2012-11-06       Impact factor: 2.396

9.  Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.

Authors:  Dolan Sondhi; Stephen M Kaminsky; Neil R Hackett; Odelya E Pagovich; Jonathan B Rosenberg; Bishnu P De; Alvin Chen; Benjamin Van de Graaf; Jason G Mezey; Grace W Mammen; Denesy Mancenido; Fang Xu; Barry Kosofsky; Kaleb Yohay; Stefan Worgall; Robert J Kaner; Mark Souwedaine; Bruce M Greenwald; Michael Kaplitt; Jonathan P Dyke; Douglas J Ballon; Linda A Heier; Szilard Kiss; Ronald G Crystal
Journal:  Sci Transl Med       Date:  2020-12-02       Impact factor: 17.956

Review 10.  Stem Cell Research Tools in Human Metabolic Disorders: An Overview.

Authors:  Serena Ricci; Pietro Cacialli
Journal:  Cells       Date:  2021-10-07       Impact factor: 6.600
  10 in total

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