beta-thalassemia
At ESHG, Researchers Present New Noninvasive Prenatal Tests, Disease Recurrence Risk Study
Scientists from the UK and China discussed two new NIPTs that include single-gene disorders and a study to estimate disease recurrence risk in families.
Horizon Discovery, Rutgers Collaborate to Commercialize CRISPR Base Editing Technology
The partners are planning to integrate the technology into Horizon's research tools and services and to develop it for applications in cell therapeutics.
Singapore Team Develops Sequencing Method to Calculate Fetal Fraction for Targeted Prenatal Tests
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The researchers plan to use the method in clinical research, designing noninvasive prenatal tests for single-gene disorders like beta thalassemia.
St. Jude Study Reshapes Hemoglobin Genetic Disorders With CRISPR/Cas9 Editing
Scientists introduced an indel to a promoter region to mimic a benign, naturally-occurring mutation that activated fetal hemoglobin production.
Scientists Edit Beta-thalassemia Gene in Human Zygotes with CRISPR/Cas9
Though the edit was made in non-viable zygotes, the study wades into human germline engineering, a topic of great consternation for the CRISPR/Cas9 research community.