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Gene delivery / Applied genetics / Medical genetics / Medical research / Molecular biology / N-Acetylaspartic acid / Canavan disease / Adeno-associated virus / Gene therapy / Biology / Health / Medicine


HUMAN GENE THERAPY 13:1391–1412 (July 20, 2002) © Mary Ann Liebert, Inc. Clinical Protocol Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene (ASPA)
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Document Date: 2009-11-06 14:09:33


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City

Worcester / /

Company

Primedica Corporation / Mary Ann Liebert Inc. / IgG / /

Continent

Europe / South America / /

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Event

FDA Phase / Natural Disaster / Product Issues / Person Communication and Meetings / /

Facility

Children’s Hospital of Philadelphia / Cooper Hospital / National Cancer Institute / Chapel Hill / /

IndustryTerm

Magnetic resonance imaging / therapy for Canavan disease / parietal site / diffusion tensor imaging / imaging / food consumption / target site / clinical protocol / Therapy of Canavan Disease / injection site / frontal lobe site / study protocol / frontal site / final clinical protocol / parental assessment tool / occipital site / /

MedicalCondition

mild respiratory distress / neurodegenerative disease / seizures / coma / truncal hypotonia / chronic vegetative state / cardiac arrhythmia / congenital heart failure / anaphylactic reactions / diarrhea / severe seizures / high fever / paralysis / mental retardation / mild post-operative fever / hyperreflexia / genetic disease / stroke / Canavan Disease / fever / mild fever / vomiting / enzymatic deficiency / monogeneic / autosomal recessive disease / Bertrand disease / infection / documented severe allergy / renal failure / mild proteinuria / severe cognitive impairment / intractable status epilepticus / headache / nausea / Tremor / cystic fibrosis / hemophilia / blindness / hypotension / ataxia / disease / infectious titer / certain infections / infectious meningitis / neurological disease / pain / brain cancer / untreated Canavan disease / severe intracranial hypertension / infectious particles / /

MedicalTreatment

surgery / gastric feeding tubes / Clinical Protocol / antibiotics / Gene Therapy / catheter / catheters / cannula / /

NaturalFeature

Chapel Hill / /

Organization

Medical School-UMDNJ and Cooper Hospital / investigators / study monitor / and independent data monitoring board / N.I.H. / office of Recombinant DNA Activities / Independent Data Monitoring Committee / UMDNJ-Robert Wood Johnson Medical School / Neurosurgical administration / Cooper Hospital / Institutional Review Committee / F.D.A. / Safety Endpoints 1Robert Wood Johnson Medical School / Children’s Hospital of Philadelphia / National Cancer Institute / Monitoring Committee / Coriell Center for Medical Research / Pennsylvania Medical School / Intracranial administration / /

Person

JOAN RUPIN / Gene Vector / PAOLA LEONE / MICHAEL GOLDBERG / ANGELIQUE CAMP / ELIZABETH SASLOW / OLGA GOLDFARB / DAVID SHERA / SCOTT MCPHEE / WILLIAM SHARRAR / EDWIN KOLODNY / JYUU WANG / JOHN HASELGROVE / LARISA LIOUTERMAN / LARISSA BILANIUK / Van Bogaert / JUDE SAMULSKI / Mullen Scales / MARK TESTAIUTI / PETER HURH / P. Leone / Cell / Robert Wood Johnson / /

Position

g.p. / neurosurgeon / referring physician / †Corresponding author / clinical coordinator / investigator / translator / neurologist and a behavioral psychologist / Director / *NINDS Principal Investigator / same neurologist / study biostatistician / reporter / /

Product

protamine sulfate / mannitol / To supplement / T1 / PO2 / to / gene / AAV-2 / /

ProvinceOrState

Pennsylvania / North Carolina / /

RadioStation

NAA / /

Region

Middle East / /

Technology

1391 1392 CLINICAL PROTOCOL / apoptosis / 1399 CLINICAL PROTOCOL / final clinical protocol / 1397 CLINICAL PROTOCOL / Magnetic resonance imaging / gene expression / spectroscopy / 1395 CLINICAL PROTOCOL / Recombinant DNA / 1398 protocol / study protocol / clinical protocol / antibodies / MRI / GENE THERAPY / gene delivery / 1400 CLINICAL PROTOCOL / 1394 CLINICAL PROTOCOL / 1396 CLINICAL PROTOCOL / /

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