New perspectives in gene therapy for inherited disorders

Maria Pia Cicalese; Alessandro Aiuti

doi:10.1111/pai.13149

New perspectives in gene therapy for inherited disorders

Maria Pia Cicalese, Alessandro Aiuti

IRCCS Ospedale San Raffaele

Research output: Contribution to journal › Article › peer-review

Abstract

Gene therapy has become promising in many fields of medicine, as a single treatment could allow long-lasting and curative benefits. New medicines based on cell gene correction are expected to occur in upcoming years and will be hopefully part of the therapeutic armamentarium for inherited and acquired diseases. Issues related to the costs of these new therapies and access to care for all patients, and procedures and expertise needed to facilitate their application worldwide require to be addressed, together with long-term safety and efficacy monitoring.

Original language	English
Pages (from-to)	5-7
Number of pages	3
Journal	Pediatric Allergy and Immunology
Volume	31
Issue number	S24
DOIs	https://doi.org/10.1111/pai.13149
Publication status	Published - Feb 1 2020

Keywords

cancer
gene therapy
immunodeficiency
inherited disorders

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Immunology and Allergy
Immunology

Access to Document

10.1111/pai.13149

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abstract = "Gene therapy has become promising in many fields of medicine, as a single treatment could allow long-lasting and curative benefits. New medicines based on cell gene correction are expected to occur in upcoming years and will be hopefully part of the therapeutic armamentarium for inherited and acquired diseases. Issues related to the costs of these new therapies and access to care for all patients, and procedures and expertise needed to facilitate their application worldwide require to be addressed, together with long-term safety and efficacy monitoring.",

keywords = "cancer, gene therapy, immunodeficiency, inherited disorders",

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