Regenerative pharmacology in the treatment of genetic diseases: the paradigm of muscular dystrophy.
Current evidence supports the therapeutic potential of pharmacological interventions that counter the progression of genetic disorders by promoting regeneration of the affected organs or tissues. The rationale behind this concept lies on the evidence that targeting key events downstream of the genetic defect can compensate, at least partially, the pathological consequence of the related disease. In this regard, the beneficial effect exerted on animal models of muscular dystrophy by pharmacological strategies that enhance muscle regeneration provides an interesting paradigm. In this review, we describe and discuss the potential targets of pharmacological strategies that promote regeneration of dystrophic muscles and alleviate the consequence of the primary genetic defect. Regenerative pharmacology provides an immediate and suitable therapeutic opportunity to slow down the decline of muscles in the present generation of dystrophic patients, with the perspective to hold them in conditions such that they could benefit of future, more definitive, therapies.
Mozzetta
Chiara
C
Dulbecco Telethon Institute at Fondazione Santa Lucia/EBRI, Via di Fosso Fiorano, 64-00143 Roma, Italy.
Minetti
Giulia
G
Puri
Pier Lorenzo
PL
D000818:Animals; D006801:Humans; D009136:Muscular Dystrophies / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D055435:Myostatin / Q000037:antagonists & inhibitors / Q000378:metabolism
D055435:Myostatin