Literature DB >> 10762513

Rapid immunologic diagnosis of classic late infantile neuronal ceroid lipofuscinosis.

Y Kurachi1, A Oka, M Mizuguchi, Y Ohkoshi, M Sasaki, M Itoh, M Hayashi, Y Goto, S Takashima.   

Abstract

OBJECTIVE: To establish a new method for rapid diagnosis of late infantile neuronal ceroid lipofuscinosis (LINCL, CLN2) using specific polyclonal antibodies against the CLN2 gene product.
METHODS: Cells and tissues were obtained from five patients with LINCL, two with variant type NCL, three with other lysosomal storage diseases, and eight control subjects. Two antibodies were raised against N- and C-terminal peptide fragments of the normal product of the CLN2 gene. The authors examined the possibility of diagnosis of LINCL with immunostaining and immunoblotting using specific antibodies made of the recently identified defective gene in LINCL.
RESULTS: Immunoreactivity with these antibodies showed the absence or marked reduction of CLN2 immunoreactivity in the lymphocytes, lymphoblasts, and fibroblasts of all five patients with LINCL examined.
CONCLUSIONS: These results indicate the usefulness of this diagnostic method based on the changes in CLN2 immunoreactivity. This relatively simple, specific, and cost-effective method is a promising diagnostic tool for this disease, although additional studies are necessary.

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Year:  2000        PMID: 10762513     DOI: 10.1212/wnl.54.8.1676

Source DB:  PubMed          Journal:  Neurology        ISSN: 0028-3878            Impact factor:   9.910


  3 in total

1.  A critical tryptophan and Ca2+ in activation and catalysis of TPPI, the enzyme deficient in classic late-infantile neuronal ceroid lipofuscinosis.

Authors:  Salomon Kuizon; Kathleen DiMaiuta; Marius Walus; Edmund C Jenkins; Marisol Kuizon; Elizabeth Kida; Adam A Golabek; Daniel O Espinoza; Raju K Pullarkat; Mohammed A Junaid
Journal:  PLoS One       Date:  2010-08-03       Impact factor: 3.752

2.  Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients.

Authors:  Sara E Mole; Angela Schulz; Eben Badoe; Samuel F Berkovic; Emily C de Los Reyes; Simon Dulz; Paul Gissen; Norberto Guelbert; Charles M Lourenco; Heather L Mason; Jonathan W Mink; Noreen Murphy; Miriam Nickel; Joffre E Olaya; Maurizio Scarpa; Ingrid E Scheffer; Alessandro Simonati; Nicola Specchio; Ina Von Löbbecke; Raymond Y Wang; Ruth E Williams
Journal:  Orphanet J Rare Dis       Date:  2021-04-21       Impact factor: 4.123

3.  Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease.

Authors:  Kevin Hammon; Greg de Hart; Brian R Vuillemenot; Derek Kennedy; Don Musson; Charles A O'Neill; Martin L Katz; Joshua W Henshaw
Journal:  Clin Transl Sci       Date:  2021-06-02       Impact factor: 4.689

  3 in total

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