Contains Nonbinding Recommendations
30
IX. REFERENCES
1. Guidance for Industry: Gene Therapy Clinical Trials – Observing Subjects for Delayed
Adverse Events, November 2006. https://www.fda.gov/media/72225/download
2. Human Genome Editing: Science, Ethics, and Governance, National Academy Press,
Washington D.C., 2017.
3. Donahue, RE, et al., Helper virus induced T cell lymphoma in nonhuman primates after
retroviral mediated gene transfer, Journal of Experimental Medicine, 1992;176:1125-
1135.
4. Hacein-Bey-Abina, S, et al., Sustained correction of X-linked severe combined
immunodeficiency by ex vivo gene therapy, N. Engl. J. Med, 2002; 346:1185-1193.
5. Biological Response Modifiers Advisory Committee (BRMAC), Meeting Minutes,
Department of Health and Human Services, Food and Drug Administration, CBER,
October 10, 2002.
6. Biological Response Modifiers Advisory Committee, Meeting Minutes Department of
Health and Human Services (BRMAC), Food and Drug Administration, CBER,
November 17, 2000; April 6, 2001; and October 24, 2001.
7. Nyberg, K, et al., Workshop on long-term follow-up of participants in human gene
transfer research, Molecular Therapy, 2004; 6:976-980.
8. Cornetta, K, et al., Screening Clinical Cell Products for Replication Competent
Retrovirus: The National Gene Vector Biorepository Experience, Mol. Ther. Methods
Clin. Dev., 2008; 10:371-378.
9. Marcucci, KT, et al., Retrovial and Lentiviral Safety Analysis of Gene-Modified T Cell
Products and Infused HIV and Oncology Patients, Mol. Ther., 2018; 26(1):269-279.
10. Hacein-Bey-Abina, S, et al., LMO2-associated clonal T cell proliferation in two patients
after gene therapy for SCID-X1, Science, 2003; 302:415-419.
11. Hacein-Bey-Abina, S, et al., Insertional oncogenesis in 4 patients after retrovirus-
mediated gene therapy of SCID-X1, J. Clin Invest, 2008; 118:3132-3142.
12. Braun, et al., Gene Therapy for Wiskott-Aldrich Syndrome—Long term Efficacy and
Genotoxicity, Science Translational Medicine, 2014; 6:227.
13. Cavazzana-Calvo, et al., Gene therapy of human severe combined immunodeficiency
(SCID)-X1 disease, Science, 2000; 288:669.
14. Howe, et al., Insertional mutagenesis combined with acquired somatic mutations causes
leukemogenesis following gene therapy of SCID-X1 patients, J Clin Invest, 2008;
118:143-150.
15. Cavazzana-Calvo, et al., Transfusion independence and HMGA2 activation after gene
therapy of human β-thalassaemia, Nature, 2010; 467:318-322.
16. Cavazzana-Calvo, et al., Haematopoietic stem cell transplantation for SCID patients:
where do we stand?, British Journal of Haematology, 2013; 160:146-152.
17. Niedere, HA and CRM Bangham, Integration site and clonal expansion in human chronic
retroviral infection and gene therapy, Viruses, 2014; 6:4140-4164.