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Medicine / Neuronal ceroid lipofuscinosis / Batten disease / Adeno-associated virus / Infantile neuronal ceroid lipofuscinosis / Epileptic seizure / Lipid storage disorders / Rare diseases / Health


Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Serotype rh.10 Expressing the Human CLN2 cDNA to Children with Late Infantile Neuronal Ceroid Lipofuscinosis NIH-OBA # [removed]
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Document Date: 2014-01-06 12:39:09

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File Size: 1,95 MB

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City

Hamburg / /

Facility

Genetic Medicine Weill Cornell Medical College / /

IndustryTerm

gc/site / /

MedicalCondition

Batten Disease / vegetative state / Late Infantile Neuronal Ceroid Lipofuscinoses / Cognitive impairment / Disease / seizures / /

MedicalTreatment

catheters / /

Organization

R. Crystal Department / Direct CNS Administration / Genetic Medicine Weill Cornell Medical College / National Institute of Health / /

Technology

Gene Therapy / MRI / /

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