Related Experiment Video
Updated: Oct 3, 2026
06:40
Mouse Fetal Liver Culture System to Dissect Target Gene Functions at the Early and Late Stages of Terminal Erythropoiesis
Published on: September 9, 2014
Flipping the fetal switch across β-hemoglobinopathies
Nishi Harwani1, Akshay Sharma1
1Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA.
Cell Stem Cell
|October 1, 2026
Abstract:
Liu et al. extend the application of transformer base editing of HBG1/2 promoters from transfusion-dependent β-thalassemia alone to one patient with sickle cell disease and three patients with geographically diverse β-thalassemia genotypes.1 The responses are meaningful, but scale, genomic surveillance, burden of cure, and access now define the translational test.
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02:18Alternative RNA Splicing
Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
There are five types of alternative RNA splicing that vary in the ways the pre-mRNA segments are removed or retained in the mature mRNA. The first...
There are five types of alternative RNA splicing that vary in the ways the pre-mRNA segments are removed or retained in the mature mRNA. The first...
02:25Genetic Lingo
An organism is diploid if it inherits two variants, or alleles, of each gene, one from each parent. These two alleles constitute the genotype for a given gene. The term genotype is also used to refer to an organism’s complete set of genes. A diploid organism with two identical alleles has a homozygous genotype, whereas two different alleles indicate a heterozygous genotype. Observable traits arising from genotypes are called phenotypes, which can also be influenced by environmental factors. An...