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Finding Safe Harbor in the Human Genome

A novel framework for identifying genomic safe harbors (GSHs) is reported in Genome Biology this week, representing a new tool for genomic engineering studies and gene therapies. GSHs are regions of the genome that can maintain transgene expression without affecting host cell function and thus offer a way to improve the safety and efficiency of genome engineering. However, only a few GSHs have been identified and finding others remains technically challenging. To address this, a team led by researchers at St. Jude Children's Research Hospital developed a method for GSH identification that integrates information from polymorphic mobile element insertions that naturally occur in human populations, epigenomic signatures, and 3D chromatin organization. They applied their approach to data from the 1000 Genomes Project and the Genotype-Tissue Expression Project to reveal 19 blood GSH candidate loci. For three of these, they demonstrated the stable expression of transgenes without alternating transcription of nearby genes in erythroid cells. The researchers also presented a computer program for knowledge-based tissue-specific GSH selection. "In combination with the fast-growing genome engineering technologies, our approach has the potential to improve the overall safety and efficiency of gene and cell-based therapy in the near future," the authors write.

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.