论文信息 - Access to gene therapy for rare diseases when commercialization is not fit for purpose - 字舞流文

Access to gene therapy for rare diseases when commercialization is not fit for purpose

A. Schambach | S. Ehl | B. Neven | F. Candotti | A. Thrasher | A. Aiuti | M. Bernardo | A. Lankester | C. Booth | R. Wynn | A. Fischer | Juan Bueren | M. Cavazzana | Michael Albert | J. Prévot | Thomas Fox | Michael Maria Ester Marina Stephan Benedicte Johan Axel Ju Albert Bernardo Cavazzana Ehl Neven Prevo | Julian Sevilla | Michael H. Albert | Julián Sevilla

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[4] A. Fischer,et al. Benefit Corporation: a path to affordable gene therapies? , 2019, Nature Medicine.

[5] L. Naldini. Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives , 2019, EMBO molecular medicine.

[6] A. Aiuti,et al. Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug. , 2017, Human gene therapy.