Clinical medical reagents for use in gene therapy; pharmaceutical preparations for use in gene therapy; therapeutic pharmaceutical compositions that include recombinant adenoassociated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy, RNA interference (RNAi), and cell therapy; pharmaceutical preparations containing adeno-associated virus (AAV) vectors and nucleic acid for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, neurodegenerative disorders, metabolic and hematologic disorders, cardiovascular, oncologic and ocular diseases

'aav':54 'acid':58 'adeno':51 'adeno-associ':50 'adenoassoci':22 'associ':52 'biolog':34,65 'cardiovascular':84 'cell':45,66 'clinic':1 'composit':18 'contain':49 'deliv':62 'diseas':73,88 'disord':76,79,83 'effect':37 'express':32 'gene':7,14,39,63 'hematolog':82 'includ':20 'interfer':42 'medic':2 'metabol':80 'name':77 'neurodegen':78 'nucleic':57 'ocular':87 'oncolog':85 'pharmaceut':9,17,47 'platform':29 'prepar':10,48 'provid':27 'raav':24 'reagent':3 'recombin':21 'rna':41 'rnai':43 'sick':74 'specif':31 'system':61 'therapeut':16,36 'therapi':8,15,40,46 'treatment':71 'use':5,12,68 'vector':25,55 'viral':23 'virus':53

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