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 http://www.medengine.com/Redeem/30E8 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
Published: Jul 17, 2017
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