Canavan-Disease-Information-Page

Canavan Disease Information Page


What research is being done?

The gene for Canavan disease has been located. Many laboratories offer prenatal screening for this disorder to populations at risk. Scientists have developed animal models for this disease and are using the models to test potential therapeutic strategies.  Three strategies are currently under investigation:  gene transfer to the brain in order to replace the mutated gene for the enzyme; metabolic therapy to provide a crucial missing metabolite (acetate); and enzyme therapy where the enzyme aspartoacylase is engineered to be able to enter the brain and is injected in the the blood stream.  Encouraging results have been obtained using these strategies.

Information from the National Library of Medicine’s MedlinePlus
Leukodystrophies
 

 

×
What research is being done?

The gene for Canavan disease has been located. Many laboratories offer prenatal screening for this disorder to populations at risk. Scientists have developed animal models for this disease and are using the models to test potential therapeutic strategies.  Three strategies are currently under investigation:  gene transfer to the brain in order to replace the mutated gene for the enzyme; metabolic therapy to provide a crucial missing metabolite (acetate); and enzyme therapy where the enzyme aspartoacylase is engineered to be able to enter the brain and is injected in the the blood stream.  Encouraging results have been obtained using these strategies.

Information from the National Library of Medicine’s MedlinePlus
Leukodystrophies
 

 

The gene for Canavan disease has been located. Many laboratories offer prenatal screening for this disorder to populations at risk. Scientists have developed animal models for this disease and are using the models to test potential therapeutic strategies.  Three strategies are currently under investigation:  gene transfer to the brain in order to replace the mutated gene for the enzyme; metabolic therapy to provide a crucial missing metabolite (acetate); and enzyme therapy where the enzyme aspartoacylase is engineered to be able to enter the brain and is injected in the the blood stream.  Encouraging results have been obtained using these strategies.

Information from the National Library of Medicine’s MedlinePlus
Leukodystrophies
 

 


Definition
Definition
Treatment
Treatment
Prognosis
Prognosis
Clinical Trials
Clinical Trials
Organizations
Organizations
Publications
Publications
P.O. Box 5823
Buffalo Grove, IL 60089
Tel: 800-833-2194
450 West End Avenue
#6A
New York, NY 10024
Tel: 212-873-4640; 877-4-CANAVAN (422-6282)
88 Route 37
New Fairfield, CT 06812
Tel: 203-746-2436
4301 Connecticut Avenue, N.W.
Suite 404
Washington, DC 20008-2369
Tel: 202-966-5557; 800-336-GENE (4363)
2001 Beacon Street
Suite 204
Boston, MA 02135
Tel: 800-90-NTSAD (906-8723)
224 North 2nd Street, Suite 2
DeKalb, IL 60115
Tel: 815-748-3211; 800-728-5483