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. 2020 Feb:31 Suppl 24:5-7.
doi: 10.1111/pai.13149.

New perspectives in gene therapy for inherited disorders

Maria Pia Cicalese  1   2 , Alessandro Aiuti  1   2   3
Affiliations

New perspectives in gene therapy for inherited disorders

Maria Pia Cicalese et al. Pediatr Allergy Immunol. 2020 Feb.

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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References

REFERENCES

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