Complete short-term correction of canine hemophilia A by in vivo gene therapy.

Article

Complete short-term correction of canine hemophilia A by in vivo gene therapy.

Blood - 15 Nov 1996

Connelly S, Mount J, Mauser A, Gardner J M, Kaleko M, McClelland A, Lothrop C D

Abstract excerpt

Hemophilia A is a severe bleeding disorder caused by a deficiency in clotting factor VIII (FVIII). A canine model that closely mimics the human disease was used to determine if an adenoviral vector expressing a human FVIII cDNA could be used to correct the hemophilia A phenotype. Within 48 hours...

Topics

Adenoviridae
Animals
Dog Diseases
Dogs
Factor VIII
Feasibility Studies
Genetic Therapy
Genetic Vectors
Hemophilia A
Humans
Phenotype
Species Specificity

Community interpretation

Discuss the evidence

Ask a question, add context or challenge an interpretation. Community activity is separate from scientific evidence strength.

discussions

replies

Citations encouraged

Claims remain attributable.

No discussion yet

Be the first to add a careful, cited interpretation.