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Homfeldt, L.

Publications and source records attributed to Homfeldt, L..

2 recordsLinked to original sources

Saturating variant analysis of TERC redefines the human telomerase reverse transcription mechanism

Genetic variation at the TERC locus, encoding the telomerase RNA component, is associated with human diseases and lifespan, but a comprehensive functional annotation of this critical non-coding RNA is lacking. Here, we performed saturating variant analysis of TERC in cells and unexpectedly redefine the human telomerase catalytic mechanism. Telomerase is understood to precisely reverse transcribe six TERC templating residues to generate GGTTAG repeats, however our screen identified dominant negative effects expected of templating bases inconsistent with this annotation. In vitro and in cells, we found human telomerase uses not six, but eight TERC residues flexibly for reverse transcription, most commonly yielding GGGTTA, but variably up to AGGGTTAG. An evolutionary change in TERC adjacent to the template explains the long-standing misannotation, reversion of which shifts the main templating register in TERC back to the one assumed for decades. Remarkably, this template-adjacent change also yields hyperactive TERC variants that rapidly lengthen telomeres when introduced into cells, including those from patients with genetic telomere diseases. By functional analysis of TERC variation in human cells, our work revises core tenets of telomerase reverse transcription and provides a new mechanistic model to inform the development of telomere-directed therapeutics.

molecular biology↗

In cellulo DNA assembly for targeted genomic integration and rearrangement in human cells

Although therapeutic genome editing holds great potential to remedy diverse inherited and acquired disorders, targeted installation of medium to large sized genomic modifications in therapeutically relevant cells remains challenging. We have developed an approach that permits DNA sequence assembly and integration in human cells leveraging CRISPR-targeted dual flap synthesis. This method, named prime assembly, allows for RNA-programmable site-specific integration of single- or double-stranded DNA fragments. Unlike homology-directed repair, prime assembly was similarly active in dividing and non-dividing cells. We applied prime assembly to perform targeted exon recoding, transgene integration, and megabase-scale rearrangements, including at therapeutically relevant loci in primary human cells. Prime assembly expands the capabilities of genome engineering by enabling the targeted integration of medium to large sized DNA sequences without relying on double-stranded DNA donors, nuclease-driven double strand breaks, or cell cycle progression.

molecular biology↗