Table 1.
Clinical Trials
 |
a
The CAG promoter designation includes the CBA and CB promoter.
Alz, Alzheimer; C, completed; Can, Canavan; Cer, cerebellum; CLN6, Batten CLN6 disease; GAN, giant axonal neuropathy; IS, immunosupressor; IT, intrathecal; IV, intravenous; LINCF, Late infantile neuronal ceroid lipofuscinosis; MLD, metachromatic leukodystrophy; max vg, maximum vector genome; min vg, minimum vector genome; MPS IIIA, mucopolysaccharidosis IIIA; MPS IIIB, mucopolysaccharidosis IIIB; Lom, lombar; N, no; NA, not available; NBM, nucleus basalis of meynert; O, ongoing; Par, Parkinson; PeV, peripheral vein; Put, Putamen; SMA I, spinal muscular atrophy type I; SN, substancia nigra; StN, subthalamique nucleus; Str, striatum; WM, white matter; Y, yes.