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Sgrizzi, S.

Publications and source records attributed to Sgrizzi, S..

3 recordsLinked to original sources

Gigabase-scale deletion scanning of the human genome

What fraction of the human genome is essential for cellular viability? To date, essentiality in human cells has been mapped almost exclusively at the level of individual open reading frames (ORFs). Whether noncoding regions and broader architectural features of the genome are required for human cells to remain viable, and where the boundaries of any such regions lie, remains largely unexplored. Here we introduce Shred-seq, which couples Type I-C CRISPR-Cas3-mediated deletions with phage polymerase-based genotyping to enable large-scale deletion scans of the human genome. Shred-seq leverages thousands of genomically integrated, mapped target sites as launchpads for Cas3 to initiate unidirectional deletions ranging in size from hundreds of base pairs (bp) to hundreds of kb. Breakpoints are directly captured at high resolution by in vitro or in situ transcription from flanking phage polymerase promoters, enabling bulk or single-cell phenotyping, respectively. In this proof-of-concept, we generate and genotype 36,257 independent deletions originating from 9,604 Cas3 launchpads, individually spanning 100 bp to 500 kb, collectively covering 461 Mb (14% of the human genome), and totaling to 2.55 Gb of deleted sequence ([~]10-fold coverage of these regions). Surviving deletions are depleted not only for essential protein-coding genes, but also for active, conserved and mutation-constrained non-coding sequences, directly quantifying purifying selection across both coding and noncoding intervals. Deletion length distributions further enable annotation-agnostic fine mapping of essential region boundaries and establish an empirical lower bound on the fraction of the human genome required for cellular viability. Finally, we demonstrate compatibility with single-cell RNA-seq (scRNA-seq), enabling direct linkage of specific deletions to transcriptional phenotypes. Together, Shred-seq provides a scalable platform for the systematic dissection of genome architecture and noncoding function, as well as for generating training data for predictive and generative models of genome structure-function, analogous to the roles that conventional Perturb-seq screens play for ORFs. By enabling the empirical delimitation of the genomic content required for human cellular viability, Shred-seq may also open a path to the construction of a minimal human genome.

genomics↗

Programmable Lipid Nanoparticle Targeting via Corona Engineering

Lipid nanoparticles (LNPs) are a versatile platform for in vivo delivery of biomolecules, yet systemically administered LNPs predominantly accumulate in the liver, limiting extrahepatic applications. This tropism arises from LNP adsorption of serum proteins, particularly apolipoprotein E (ApoE), which binds to LDL receptors (LDLR) on hepatocytes. Here, we overcome this tropism with two compatible strategies. First, we engineer dead ApoE mutants (dApoE) with five receptor-binding domain substitutions that selectively disrupt the ApoE-LDLR interaction but retain lipid binding. In cultured cells, pre-coating with these dApoE markedly inhibited LDLR-mediated uptake. Second, we pretreat cells with hyperactive PCSK9 (haPCSK9) to internalize surface LDLR, similarly reducing the LDLR-mediate uptake of LNPs. In vivo, both strategies substantially reduced liver LNP transduction without inducing redistribution to other major organs. To retarget LNP to new cell types we combined antibody conjugation with dApoE or haPCSK9, effectively engineering tropism to T cells, brain and lung tissues in vivo with substantially reduced hepatic background. In pilot studies, this strategy enabled specific delivery of reporter mRNAs to additional tissues, including megakaryocytes, hematopoietic progenitor cells, and cardiac tissue, and in aged T cells, to deliver miRNA cargos that produced a sustained reduction in DNA damage markers following a single systemic dose. dApoE coated CD5-targeted LNPs generated CAR+ T cells that retained cytotoxicity against CD19+ targets, while simultaneously reducing hepatocyte transduction by 90%. These findings establish a modular framework that integrates dApoE and haPCSK9-mediated detargeting with antibody-based retargeting, allowing for improvements in LNP specificity and broadening the therapeutic scope of LNPs.

bioengineering↗

Programmable RNA-guided endonucleases are widespread in eukaryotes and their viruses

TnpB proteins are RNA-guided nucleases that are broadly associated with IS200/605 family transposons in prokaryotes. TnpB homologs, named Fanzors, have been detected in genomes of some eukaryotes and large viruses, but their activity and functions in eukaryotes remain unknown. We searched genomes of diverse eukaryotes and their viruses for TnpB homologs and identified numerous putative RNA-guided nucleases that are often associated with various transposases, suggesting they are encoded in mobile genetic elements. Reconstruction of the evolution of these nucleases, which we rename Horizontally-transferred Eukaryotic RNA-guided Mobile Element Systems (HERMES), revealed multiple acquisitions of TnpBs by eukaryotes and subsequent diversification. In their adaptation and spread in eukaryotes, HERMES proteins acquired nuclear localization signals, and genes captured introns, indicating extensive, long term adaptation to functioning in eukaryotic cells. Biochemical and cellular evidence show that HERMES employ non-coding RNAs encoded adjacent to the nuclease for RNA-guided cleavage of double-stranded DNA. HERMES nucleases contain a re-arranged catalytic site of the RuvC domain, similar to a distinct subset of TnpBs, and lack collateral cleavage activity. We demonstrate that HERMES can be harnessed for genome editing in human cells, highlighting the potential of these widespread eukaryotic RNA-guided nucleases for biotechnology applications.

molecular biology↗