Harrington Rhodes (quartbeast20)

Thalassemia and hemophilia are common inherited blood disorders caused by genetic abnormalities. These diseases are difficult to cure and can be inherited to the next generation, causing severe family and social burden. The emergence of gene therapy provides a new treatment for genetic diseases. However, since its first clinical trial in 1990, the development of gene therapy has not been as optimistic in the past three decades as one could hope. The development of gene-editing technology, particularly the third generation gene-editing technology CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9), has given hope in such therapeutic approach for having advantages in high editing efficiency, simple operation, and low cost. Gene editing-mediated gene therapy has thus received increasing attention from the biomedical community. It has shown promises for the treatment of inherited blood disorders, such as thalassemia and hemophilia. This paper reviews the fundamental research progress of gene therapy for β-thalassemia and hemophilia based on CRISPR/Cas9 technology in the past six years. It also summarizes the CRISPR/Cas9-based clinical trials of gene therapy. The problems and possible solutions to this technology for gene therapy are also discussed, thereby providing a reference for the research on gene therapy of inherited blood disorders based on CRISPR/Cas9 technology.Knowledge of the origin of eukaryotes is key to broadening our understanding of the eukaryotic genome and the relationship among internal structures within a eukaryotic cell. Since the discovery of archaea in 1977 and the proposal of three-domain tree of life by the American microbiologist Carl Woese, the intimate relationship in evolution between eukaryotes and archaea has been demonstrated by considerable experiments and analyses. From the beginning of the 21st century, with the development of phylogenetic methods and the discovery of new archaeal phyla more related to eukaryotes, increasing evidence has shown that Eukarya and Archaea should be merged into one domain, leading to a two-domain tree of life. Nowadays, the Asgard superphylum discovered via metagenomic analysis is regarded as the closest prokaryotes to eukaryotes. Nevertheless, several key questions are still under debate, such as what the ancestors of the eukaryotes were and when mitochondria emerged. Here, we review the current research progress regarding the changes of the tree of life and the detailed eukaryotic evolutionary mechanism. We show that the recent findings have greatly improved our knowledge on the origin of eukaryotes, which will pave the way for future studies.Medical Genetics bridges basic research and clinical studies. It is one of the important basic courses for medical students. To cultivate students' self-regulated learning ability and improve the teaching quality of Medical Genetics, a presentation-assimilation-discussion (PAD) class mode is adopted in the teaching process. In the teaching setup, we gave students the proceedings and time guarantee for self-regulated learning, and guided the students through teaching, discussion and homework for their independent learning. The resulting evaluation of "Self-Regulated Learning Scale for College Students" shows that the students' learning motivation and learning strategy are enhanced compared with those before the implementation of PAD teaching, the total score of the evaluation is significantly increased, the students' self-regulated learning ability is improved, and satisfactory teaching results are achieved. Selleckchem EKI-785 We conclude that PAD class could contribute to students' self-regulated learning.The processes of self-renewal and differentiation of germ cells are crucial to the development of male infertility and germ cell tumors. CG8005 gene is one of the regulatory factors of the testicular germ stem cells in Drosophila melanogaster, and its functional mechanism is