Exploring Novel Clinical Trial Designs for Gene-Based Therapies Proceedings of a Workshop (2020) / Chapter Skim
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References
Pages 77-82
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2018. Hematopoietic stem cell transplantation for adult sickle cell disease in the era of universal donor availability.
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2003. Neuromuscular defects in a Drosophila survival motor neuron gene mutant.
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2017. Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.
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2018. Correction of biochemical abnormalities and improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease.
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2019. A CRISPR focus on attitudes and beliefs toward somatic genome editing from stakeholders within the sickle cell disease community.
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2018. DMD genotype correlations from the Duchenne registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype.
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