Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease.

Leone, P

Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease. [electronic resource] - Current opinion in molecular therapeutics Aug 1999 - 487-92 p. digital

Publication Type: Journal Article; Review

1464-8431


Acetates--therapeutic use
Acetazolamide--therapeutic use
Amidohydrolases--deficiency
Animals
Aspartic Acid--analogs & derivatives
Brain--enzymology
Brain Edema--etiology
Calcium Compounds
Canavan Disease--diagnosis
Child
Child, Preschool
Cholesterol--administration & dosage
Combined Modality Therapy
Cytomegalovirus--genetics
DNA, Recombinant--administration & dosage
Dependovirus--genetics
Female
Fetal Diseases--diagnosis
Genetic Therapy
Genetic Vectors--administration & dosage
Glycerophospholipids--administration & dosage
Humans
Infant
Injections, Intraventricular
Liposomes--administration & dosage
Male
Mitochondria--metabolism
Nerve Tissue Proteins--deficiency
Osmotic Pressure
Phosphatidylethanolamines
Prenatal Diagnosis
Primates
Promoter Regions, Genetic
Protamines--administration & dosage
Rats