Literature DB >> 11589001

Cardiac pathology in neuronal ceroid lipofuscinoses--a clinicopathologic correlation in three patients.

I L Hofman1, A C van der Wal, K P Dingemans, A E Becker.   

Abstract

We report the clinical details and the pathology of the heart at autopsy of three neuronal ceroid lipofuscinosis (NCL) patients. Two patients were diagnosed as classical juvenile NCL and one as a variant juvenile NCL (JNCL) with granular osmiophilic deposits (GRODs). Cardiac findings during life were retrospectively evaluated and included left ventricular hypertrophy with repolarization disturbances (ECG findings) in two patients with classical JNCL and severe bradycardia with periods of sinus arrest in one of them, severe supraventricular tachycardias during anaesthesia in the variant JNCL-patient. At autopsy myocardial and valvular storage of lipopigments, diagnostic for NCL, was observed histologically and confirmed ultrastructurally in all three cases. In two patients with JNCL the storage was associated with hypertrophy and dilation of both ventricles, degenerative myocardial changes, interstitial fibrosis and fatty replacement. Abundant accumulation and degeneration were seen in all components of the conduction system in three patients, which outreached at several places by far the storage of the adjacent myocardium. Our observations indicate a prominent involvement of the heart in NCL, with preference of storage for the conduction system of the heart.

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Year:  2001        PMID: 11589001     DOI: 10.1053/ejpn.2000.0465

Source DB:  PubMed          Journal:  Eur J Paediatr Neurol        ISSN: 1090-3798            Impact factor:   3.140


  19 in total

1.  Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis.

Authors:  Marie S Roberts; Shannon L Macauley; Andrew M Wong; Denis Yilmas; Sarah Hohm; Jonathan D Cooper; Mark S Sands
Journal:  J Inherit Metab Dis       Date:  2012-02-07       Impact factor: 4.982

2.  Analysis of potential biomarkers and modifier genes affecting the clinical course of CLN3 disease.

Authors:  Anne-Hélène Lebrun; Parisa Moll-Khosrawi; Sandra Pohl; Georgia Makrypidi; Stephan Storch; Dirk Kilian; Thomas Streichert; Benjamin Otto; Sara E Mole; Kurt Ullrich; Susan Cotman; Alfried Kohlschütter; Thomas Braulke; Angela Schulz
Journal:  Mol Med       Date:  2011-08-18       Impact factor: 6.354

3.  Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis.

Authors:  Jie Hu; Jui-Yun Lu; Andrew M S Wong; Linda S Hynan; Shari G Birnbaum; Denis S Yilmaz; Barbara M Streit; Ewelina M Lenartowicz; Thomas C M Thompson; Jonathan D Cooper; Sandra L Hofmann
Journal:  Mol Genet Metab       Date:  2012-05-22       Impact factor: 4.797

4.  The juvenile Batten disease protein, CLN3, and its role in regulating anterograde and retrograde post-Golgi trafficking.

Authors:  Susan L Cotman; John F Staropoli
Journal:  Clin Lipidol       Date:  2012-02

5.  A murine model of infantile neuronal ceroid lipofuscinosis-ultrastructural evaluation of storage in the central nervous system and viscera.

Authors:  Nancy Galvin; Carole Vogler; Beth Levy; Attila Kovacs; Megan Griffey; Mark S Sands
Journal:  Pediatr Dev Pathol       Date:  2007-05-23

6.  Children with infantile neuronal ceroid lipofuscinosis have an increased risk of hypothermia and bradycardia during anesthesia.

Authors:  Ning Miao; Sondra W Levin; Eva H Baker; Rafael C Caruso; Zhongjian Zhang; Andrea Gropman; Deloris Koziol; Robert Wesley; Anil B Mukherjee; Zenaide M N Quezado
Journal:  Anesth Analg       Date:  2009-08       Impact factor: 5.108

Review 7.  Vision loss in juvenile neuronal ceroid lipofuscinosis (CLN3 disease).

Authors:  Madhu M Ouseph; Mark E Kleinman; Qing Jun Wang
Journal:  Ann N Y Acad Sci       Date:  2016-01-08       Impact factor: 5.691

8.  Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis.

Authors:  Jui-Yun Lu; Jie Hu; Sandra L Hofmann
Journal:  Mol Genet Metab       Date:  2009-12-05       Impact factor: 4.797

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.  Loss of CLN3, the gene mutated in juvenile neuronal ceroid lipofuscinosis, leads to metabolic impairment and autophagy induction in retinal pigment epithelium.

Authors:  Yu Zhong; Kabhilan Mohan; Jinpeng Liu; Ahmad Al-Attar; Penghui Lin; Robert M Flight; Qiushi Sun; Marc O Warmoes; Rahul R Deshpande; Huijuan Liu; Kyung Sik Jung; Mihail I Mitov; Nianwei Lin; D Allan Butterfield; Shuyan Lu; Jinze Liu; Hunter N B Moseley; Teresa W M Fan; Mark E Kleinman; Qing Jun Wang
Journal:  Biochim Biophys Acta Mol Basis Dis       Date:  2020-06-25       Impact factor: 6.633

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