Fig. 1
From: Gene therapy for ultrarare diseases: a geneticist’s perspective
Application of gene therapy for ultrarare diseases. CGD chronic granulomatous disease, EDS Ehlers–Danos syndrome, EE epileptic encephalopathy, ML mucolipidoses, MPS mucopolysaccharidoses, NBIA neurodegeneration with brain iron accumulation, OA organic acidurias, OI osteogenesis imperfecta, SCID severe combined immunodeficiency, SED spondyloepiphyseal dysplasia, neurotransmitter deficiency, UCD urea cycle disorders