Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease

Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease Adv Ther (2017) 34:2007–2021 DOI 10.1007/s12325-017-0587-7 ORIGINAL RESEARCH Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease . . . Heather Strong Monica J. Mitchell Alana Goldstein-Leever . . Lisa Shook Punam Malik Lori E. Crosby Received: October 19, 2016 / Published online: July 17, 2017 Springer Healthcare Ltd. 2017 transplantation, the only curative treatment, is ABSTRACT available to less than 20% of patients because of a number of access barriers. Gene transfer ther- Introduction: Sickle cell disease (SCD) is a apy (GTT) has been shown to be curative in chronic genetic disease with high morbidity animal models and is approved for use in and early mortality; it affects nearly 100,000 humans for early-phase studies at a few centers. individuals in the USA. Bone marrow GTT would offer a more accessible treatment option available to all patients. It is important Punam Malik and Lori E. Crosby are co-senior authors. to understand patient perspectives on GTT to help ensure human clinical trial success. Enhanced content To view enhanced content for this article go to Methods: Two focus groups were conducted F060677D333B. with younger (18–30 years) and older (31 years and older) adults with SCD to obtain data on Advances in Therapy Springer Journals

Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease

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Springer Healthcare
Copyright © 2017 by Springer Healthcare Ltd.
Medicine & Public Health; Internal Medicine; Oncology; Cardiology; Rheumatology; Endocrinology; Pharmacology/Toxicology
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