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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.