Pharmaceutical preparations for treatment of rare genetic diseases; pharmaceutical preparations primarily comprising cells for treatment of rare diseases; pharmaceutical preparations and therapeutic agents for use in gene therapy for treatment of rare diseases; pharmaceutical preparations for gene therapy for the treatment of genetic diseases or disorders; pharmaceutical preparations for use in the treatment of gene therapy treatable diseases.

'agent':23 'cell':13 'compris':12 'diseas':8,18,33,44,58 'disord':46 'gene':27,37,55 'genet':7,43 'pharmaceut':1,9,19,34,47 'prepar':2,10,20,35,48 'primarili':11 'rare':6,17,32 'therapeut':22 'therapi':28,38,56 'treatabl':57 'treatment':4,15,30,41,53 'use':25,50

Medical and scientific research in the field of gene therapy; medical and scientific research in the field of gene editing for developing cells and pharmaceuticals for medical and clinical use; Research and development of pharmaceuticals using gene editing

'cell':23 'clinic':29 'develop':22,33 'edit':20,38 'field':7,17 'gene':9,19,37 'medic':1,11,27 'pharmaceut':25,35 'research':4,14,31 'scientif':3,13 'therapi':10 'use':30,36

SANGRAIL

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