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Therapeutic gene editing strategies using CRISPR-Cas9 for theβ-hemoglobinopathies

The Journal of Biomedical Research 2021年第2期35卷 115-134页

作者： James B.Papizan Shaina N.Porter Akshay Sharma shondra m.pruett-miller Department of Cellular and molecular Biology St.Jude Children's Research HospitalMemphisTN 38105USA Center for Advanced Genome Engineering St.Jude Children's Research HospitalMemphisTN 38105USA Department of Bone marrow Transplantation and Cellular Therapy St.Jude Children's Research HospitalMemphisTN 38105USA

With advancements in gene editing technologies,our ability to make precise and efficient modifications to the genome is increasing at a remarkable rate,paving the way for scientists and clinicians to uniquely treat a multitude of previously irremediable ***-Cas9,short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9,is a gene editing platform with the ability to alter the nucleotide sequence of the genome in living *** technology is increasing the number and pace at which new gene editing treatments for genetic disorders are moving toward the ***β-hemoglobinopathies are a group of monogenic diseases,which despite their high prevalence and chronic debilitating nature,continue to have few therapeutic options *** this review,we will discuss our existing comprehension of the genetics and current state of treatment forβ-hemoglobinopathies,consider potential genome editing therapeutic strategies,and provide an overview of the current state of clinical trials using CRISPR-Cas9 gene editing.

关键词： sickle cell disease sickle cell anemia fetal hemoglobin hemoglobinopathy CRISPR gene editing genome engineering

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The F-box-only protein 44 regulates pregnane X receptor protein level by ubiquitination and degradation

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Acta Pharmaceutica Sinica B 2023年第11期13卷 4523-4534页

作者： Rebecca R.Florke Gee Andrew D.Huber Jing Wu Richa Bajpai Allister J.Loughran shondra m.pruett-miller Taosheng Chen Department of Chemical Biology and Therapeutics St.Jude Children's Research HospitalMemphisTN 38105USA Graduate School of Biomedical Sciences St.Jude Children's Research HospitalMemphisTN 38105USA Center for Advanced Genome Engineering and Department of Cell and molecular Biology St.Jude Children's Research HospitalMemphisTN 38105USA

Pregnane X receptor(PXR)is a ligand-activated nuclear receptor that transcriptionally upregulates drug-metabolizing enzymes[e.g.,cytochrome P4503A4(CYP3A4)]and *** the regulation of PXR target genes is well-characterized,less is known about the regulation of PXR protein *** screening an RNAi library,we identified the F-box-only protein 44(FBXO44)as a novel E3ligase for *** abundance increases upon knockdown of FBXO44,and,inversely,decreases upon overexpression of *** analysis revealed that FBXO44 interacts with PXR,leading to its ubiquitination and proteasomal degradation,and we determined that the F-box associated domain of FBXO44 and the ligand binding domain of PXR are required for the functional *** summary,FBXO44 regulates PXR protein abundance,which has downstream consequences for CYP3A4 levels and drug-drug *** results of this study provide new insight into the molecular mechanisms that regulate PXR protein level and activity and suggest the importance of considering how modulating E3ubiquitin ligase activities will affect PXR-mediated drug metabolism.

关键词： PXR FBXO44 Ubiquitination Proteasomal degradation CYP3A4 Drug-drug interactions E3 ligase Nuclear receptor

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Toxicological Evaluation of Occidiofungin against mice and Human Cancer Cell Lines

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Pharmacology & Pharmacy 2014年第11期5卷 1085-1093页

作者： Steven Lai Hing Akshaya Ravichandran Jerome Escano Jim Cooley Frank Austin Shi-En Lu Stephen pruett Leif Smith Department of Biological Sciences Texas A&M University College Station USA Department of Pathobiology and Population medicine College of Veterinary Medicine Mississippi State University USA Department of Biochemistry Molecular Biology Entomology and Plant Pathology Mississippi State University USA Department of Basic Sciences College of Veterinary Medicine Mississippi State University USA

Occidiofungin, a glyco-lipopeptide obtained from the liquid culture of Burkholderia contaminans mS14, has been identified as a novel fungicide. The natural product was shown to have a minimal amount of toxicity in a previous mouse toxicity study following intraperitoneal and subcutaneous administration. In this study, the toxicity of occidiofungin was evaluated following a 5 mg/kg intravenous tail vein injection. In addition, the toxicity of occidiofungin was evaluated against human fibroblast and cancer cell lines. Weight loss was the most significant observation following intravenous administration of occidiofungin. Histology, hematology, and blood serum chemistry did not reveal any significant signs of toxicity. The activity observed in the in vitro cytotoxicity assay against the cancer cell lines was all below 75 nm concentration of occidiofungin. To date, the potency of occidiofungin against these cancer cell lines is greater than any activity observed against fungi. The findings in this study support the need to further evaluate occidiofungin’s chemotherapeutic potential.

关键词： Antifungal Chemotherapeutic Toxicity Histopathology

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