Disclosed herein are methods of treating Canavan disease in a subject through restoring ASPA enzymatic activities in the subject by expressing exogenous wild type ASPA gene in the brain of the subject. Also disclosed are a process of producing neural precursor cells, including NPCs, glial progenitor...
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Background: Canavan disease is a rare leukodystrophy with no current treatment. rAAV-ASPA has been developed for gene delivery to the central nervous syste... McPhee,SWJ,Janson,... - 《Journal of Gene Medicine》 被引量: 448发表: 2006年 Clinical protocol. Gene therapy of Canavan disease: AA...
CHAPTER 23 – Clinical Trials of Gene Therapy for Canavan DiseaseThis chapter discusses the canavan disease (CD) treatment options using gene therapy, especially focusing on the latest results in the context of aspartoacylase (ASPA) gene transfer in human, mouse, and rat. CD is an autosomal...
Lithium citrate as treatment of Canavan disease. Canavan disease is a rare autosomal recessive leukodystrophy characterized by abnormal accumulation of N-acetyl aspartate (NAA) in brain white matter. Curr... MD Edo Solsona,LL Fernández,EM Boquet,... - 《Clinical Neuropharmacology》 被引量: 38发表...
Prognosis for Canavan disease is poor. The National Institute of Neurological Disorders and Stroke noted that usually, individuals with the disease die before age 10. However, some individuals survive into their twenties. In terms of treatment, there is no standard course, and it tends to target...
administration of rAAV9-CB6-ASPA provides a safe and effective therapeutic strategy in an open-label, individual-patient, expanded-access trial for Canavan disease. Immunomodulation was given prophylactically prior to adeno-associated virus (AAV) treatment to prevent an immune response to ASPA or ...
Canavan in 1931 described spongy degeneration of the brain in a child who was thought to have had Schilder's disease. Since that classic histological description, Canavan disease has become a distinct clinical entity, with the recognition by Van Bogaert and Bertrand that this is an autosomal reces...
There is no treatment for CD and the only prevention is through genetic counselling and prenatal diagnosis. We experienced a case of Canavan disease that was presented with hypotonia and developmental delay. Diagnosis was confirmed histologically. Radiologic findings are extensive high signal throughout...
sdisease(CD)isafatalautosomal recessivepediatricleukodystrophyinwhichpatientsshow severeneurodegenerationandtypicallydiebytheageof 10,thoughlifeexpectancyinpatientscanbehighly variable.Currently,thereisnoeffectivetreatmentforCD; however,genetherapyseemstobeafeasibleapproachto combatthedisease.Beingamonogenicdefect,the...