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Publication
Gene therapy: A new hope in sickle cell disease treatment
Journal of Pediatric Health Care -
News
Using a Family Advisory Council to Understand the Informational Needs of Prospective Gene Therapy Trial Participants: An Interview with the Co-PIs of the SCDGENE CERA Working Group
Sickle cell disease (SCD), characterized by severe pain crisis and progressive organ damage as well as early death, is one of the most common inherited blood disorders in the world. However, since 90…- sickle cell disease
- gene therapy
- Sickle Cell
- gene therapies
- community based participatory research
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Publication
Haemophilia gene therapy: Experiences and lessons from treated patients
Orphanet Journal of Rare Diseases -
Publication
Gene-targeted therapies: Towards equitable development, diagnosis, and access
American Journal of Medical Genetics Part C: Seminars in Medical Genetics -
Publication
A room with a view (and with a gene therapy drug): Gene therapy medicinal products and genetic tourism in Europe
European Journal of Health Law -
Publication
Addressing the dark matter of gene therapy: Technical and ethical barriers to clinical application
Human Genetics -
Publication
Redefining informed consent form in cell and gene therapy trials
Perspectives in Clinical Research -
Publication
Preparing newborn screening for the future: A collaborative stakeholder engagement exploring challenges and opportunities to modernizing the newborn screening system
BMC PediatricsThe future of newborn screening (NBS) will depend on the ability of the current system to adapt to increased demands resulting from advances in gene therapies. Andrews and colleagues convened NBS… -
Publication
Assessing the costs and cost-effectiveness of genomic sequencing
J Pers MedClinical Sequencing Evidence-Generating Research (CSER) Consortium. Grant number(s): U01-HG006500, U19-HD077671, F32-HG006993