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Table 1 Clinical trials with AAV-based vectors [22].

From: The state of the art of adeno-associated virus-based vectors in gene therapy

Category/Disease Clinical trial
  Phase I O/C* Phase I/II O/C Phase II O/C Phase III O/C Total
Infeccious      
   HIV vaccine 2/0 0/0 0/0 0/0 2
Cancer      
   Malignant melanoma 1/0 0/0 0/0 0/0 1
   Prostate cancer 0/1 2/1 0/0 4/0 8
   Hormone refractory prostate cancer 1/0 0/0 0/0 0/0 1
   Metastatic prostate cancer 0/0 0/0 0/0 1/0 1
Genetic      
   Lipoprotein lipase deficience 0/0 1/0 0/0 0/0 1
   Haemophilia B 1/2 0/0 0/0 0/0 3
   Early onset retinal degeneration 3/0 0/0 0/0 0/0 3
   Cystic fibrosis 2/3 1/1 0/2 0/0 9
   Muscular dystrophy 1/0 0/0 0/0 0/0 1
   Canavan disease 1/0 0/0 0/0 0/0 1
   Duchenne muscular dystrophy 1/1 0/0 0/0 0/0 2
   Limb Girdle muscular dystrophy 1/0 0/0 0/0 0/0 1
   Amyotrophic lateral sclerosis 1**/0 0/0 0/0 0/0 1
   Inherited autosomal recessive alpha-1-antitrypsin deficiency 2/0 0/0 0/0 0/0 2
   Late infantile neuronal ceroid lipofuscinosis 1/0 0/0 0/0 0/0 1
Neurological      
   Parkinson's disease 1/1 0/0 1/0 0/0 3
   Alzheimer's disease 0/0 0/1 0/0 0/0 1
   Epilepsy 1/0 0/0 0/0 0/0 1
Cardiovascular      
   Heart failure 2/0 0/0 0/0 0/0 2
Others      
   Rheumatoid arthritis 2/0 0/0 0/0 0/0 2
TOTAL 24/8 4/3 1/2 5/0 47
  1. * O/C, open/closed; ** under review.