Aggiornamento
Medicina di precisione: miti e realtŕ
Precision Medicine: what paediatricians should know
Stefano Amoroso1, Francesco Baldo1, Laura Bettini2, Benedetta Bossini1, Silvia Nucera2
1IRCCS Materno-Infantile “Burlo Garofolo”, Universitŕ di Trieste
2Universitŕ di Milano-Bicocca
Gennaio 2021 - pagg. 18 -24 | DOI: 10.53126/MEB40018
Abstract
Precision medicine is an emerging framework to maximise therapeutic benefit by modifying individual patients’ treatment considering the variations in their demographic, genomic and environmental factors. The present review describes recent advances, possible applications and unique challenges in precision medicine for some paediatric diseases. Precision medicine is no longer a distant vision; it is becoming part of the rapidly evolving present. Paediatricians should ask the right questions and give the right answers on this topic.
Riassunto
La Medicina di precisione ha oggi applicazioni concrete anche in Pediatria. Ne sono un esempio le cosiddette target therapy con l’uso di farmaci diretti contro il meccanismo molecolare della malattia, l’utilizzo sempre piů frequente dell’esoma e di tutte le scienze omiche (farmacogenomica, trascrittomica, proteomica, metabolomica) in ambito clinico, la terapia genica e la rivoluzione data in ambito emato-oncologico dalle cellule CAR-T, argomenti su cui proveremo a fare chiarezza in questo articolo. Di certo il futuro della Medicina di precisione si prospetta anche irto di difficoltŕ e contraddizioni da risolvere in primo luogo per quanto riguarda i costi e la possibilitŕ di un suo equo utilizzo. Ma č altrettanto certo che la Pediatria (fatta da tutti i pediatri) non deve farsi trovare impreparata: sia che si tratti di saper porre le giuste domande, sia che si tratti di sapere orientare delle giuste risposte.
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Classificazione MeSH
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Corrispondenza: stefanoamoroso1234@gmail.com