Designing and Testing the Activities of TAL Effector Nucleases.

Methods in molecular biology (Clifton, N.J.) (Impact Factor: 1.29). 02/2014; 1114:203-19. DOI: 10.1007/978-1-62703-761-7_13
Source: PubMed


Transcription activator-like effector nucleases (TALENs) have rapidly developed into a powerful tool for genome editing. To avoid labor-intensive and time-consuming experimental screening for active TALENs, a scoring system can help select optimal target sites. Here we describe a procedure to design active TALENs using a scoring system named Scoring Algorithm for Predicted TALEN Activity (SAPTA) and a method to test the activity of individual and pairs of TALENs.

Download full-text


Available from: Thomas James Cradick, Jan 05, 2015
  • [Show abstract] [Hide abstract]
    ABSTRACT: Introduction: β-thalassemias are caused by nearly 300 mutations of the β-globin gene, leading to low or absent production of adult hemoglobin. Achievements have been recently obtained on innovative therapeutic strategies for β-thalassemias, based on studies focusing on the transcriptional regulation of the γ-globin genes, epigenetic mechanisms governing erythroid differentiation, gene therapy and genetic correction of the mutations. Areas covered: The objective of this review is to describe recently published approaches (the review covers the years 2011 - 2014) useful for the development of novel therapeutic strategies for the treatment of β-thalassemia. Expert opinion: Modification of β-globin gene expression in β-thalassemia cells was achieved by gene therapy (eventually in combination with induction of fetal hemoglobin [HbF]) and correction of the mutated β-globin gene. Based on recent areas of progress in understanding the control of γ-globin gene expression, novel strategies for inducing HbF have been proposed. Furthermore, the identification of microRNAs involved in erythroid differentiation and HbF production opens novel options for developing therapeutic approaches for β-thalassemia and sickle-cell anemia.
    Expert Opinion on Biological Therapy 06/2014; 14(10):1-12. DOI:10.1517/14712598.2014.927434 · 3.74 Impact Factor