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Frenkel, M.

Publications and source records attributed to Frenkel, M..

4 recordsLinked to original sources

Systematic mapping of chromatin dysregulation driven by viral transcriptional regulators at scale

Viral transcriptional regulators (vTRs) reprogram host cell transcriptional and epigenetic networks to promote infection, persistence, and oncogenic transformation. Despite their broad essentiality to viral pathogenesis, thousands of putative vTRs remain functionally uncharacterized. Here we apply PROD-ATAC, a pooled single-cell epigenomic screening platform, to map chromatin accessibility dysregulation induced by over 100 vTRs in a single assay. Our screen identified dozens of vTRs which directly and indirectly remodel chromatin, including variants with no previously reported epigenetic activity. Comparative analyses revealed that unrelated vTRs frequently opened chromatin regions associated with the same host transcription factor networks, including the NF-{kappa}B, AP-1, p53, and Sp1/KLF families. Integration of chromatin accessibility and transcriptomic datasets highlighted that epigenetic perturbations often coincide with downstream gene expression changes. By adapting PROD-ATAC to incorporate small-molecule perturbations, we demonstrate that targeted inhibition of host features (including the MAPK signaling cascade) can illuminate host dependencies for vTR-induced epigenomic dysregulation. These results establish a scalable framework for discovering virus-host chromatin interactions and suggest that epigenetic manipulation of host regulatory networks is a widespread and conserved function of many diverse vTRs.

genomics↗

Highly multiplexed design of an allosteric transcription factor to sense novel ligands

Allosteric transcription factors (aTF), widely used as biosensors, have proven challenging to design for detecting novel molecules because mutation of ligand-binding residues often disrupts allostery. We developed Sensor-seq, a high-throughput platform to design and identify aTF biosensors that bind to non-native ligands. We screened a library of 17,737 variants of the aTF TtgR, a regulator of a multidrug exporter, against six non-native ligands of diverse chemical structures - four derivatives of the cancer therapeutic tamoxifen, the antimalarial drug quinine, and the opiate analog naltrexone - as well as two native flavonoid ligands, naringenin and phloretin. Sensor-seq identified novel biosensors for each of these ligands with high dynamic range and diverse specificity profiles. The structure of a naltrexone-bound design showed shape-complementary methionine-aromatic interactions driving ligand specificity. To demonstrate practical utility, we developed cell-free detection systems for naltrexone and quinine. Sensor-seq enables rapid, scalable design of new biosensors, overcoming constraints of natural biosensors.

synthetic biology↗

Discovering chromatin dysregulation induced by protein-coding perturbations at scale

Although population-scale databases have expanded to millions of protein-coding variants, insight into variant mechanisms has not kept pace. We present PROD-ATAC, a high-throughput method for discovering the effects of protein-coding variants on chromatin. A pooled library of variants is expressed in a disease-agnostic cell line, and single-cell ATAC resolves each variants effect on chromatin. Using PROD-ATAC, we characterized the effects of >100 oncofusions (a class of cancer-causing chimeric proteins) and controls and revealed that pioneer activity is a common feature of fusions spanning an enormous range of fusion frequencies. Further, fusion-induced dysregulation can be context-agnostic as observed mechanisms often overlapped with cancer and cell-type specific prior knowledge. We also showed that gain-of-function pioneering is common among oncofusions. This work provides a global view of fusion-induced chromatin. We uncovered convergent mechanisms among disparate oncofusions and shared modes of dysregulation across different cancers. PROD-ATAC is generalizable to any set of protein-coding variants.

genomics↗

Novel, provable algorithms for efficient ensemble-based computational protein design and their application to the redesign of the c-Raf-RBD:KRas protein-protein interface

The K* algorithm provably approximates partition functions for a set of states (e.g., protein, ligand, and protein-ligand complex) to a user-specified accuracy{varepsilon} . Often, reaching an{varepsilon} -approximation for a particular set of partition functions takes a prohibitive amount of time and space. To alleviate some of this cost, we introduce two algorithms into the osprey suite for protein design: O_SCPLOWFRIESC_SCPLOW, a Fast Removal of Inadequately Energied Sequences, and EWAK*, an Energy Window Approximation to K*. In combination, these algorithms provably retain calculational accuracy while limiting the input sequence space and the conformations included in each partition function calculation to only the most energetically favorable. This combined approach leads to significant speed-ups compared to the previous state-of-the-art multi-sequence algorithm, BBK*. As a proof of concept, we used these new algorithms to redesign the protein-protein interface (PPI) of the c-Raf-RBD:KRas complex. The Ras-binding domain of the protein kinase c-Raf (c-Raf-RBD) is the tightest known binder of KRas, a historically \"undruggable\" protein implicated in difficult-to-treat cancers including pancreatic ductal adenocarcinoma (PDAC). O_SCPLOWFRIESC_SCPLOW/EWAK* accurately retrospectively predicted the effect of 38 out of 41 different sets of mutations in the PPI of the c-Raf-RBD:KRas complex. Notably, these mutations include mutations whose effect had previously been incorrectly predicted using other computational methods. Next, we used O_SCPLOWFRIESC_SCPLOW/EWAK* for prospective design and discovered a novel point mutation that improves binding of c-Raf-RBD to KRas in its active, GTP-bound state (KRasGTP). We combined this new mutation with two previously reported mutations (which were also highly-ranked by O_SCPLOWOSPREYC_SCPLOW) to create a new variant of c-Raf-RBD, c-Raf-RBD(RKY). O_SCPLOWFRIESC_SCPLOW/EWAK* in O_SCPLOWOSPREYC_SCPLOW computationally predicted that this new variant would bind even more tightly than the previous best-binding variant, c-Raf-RBD(RK). We measured the binding affinity of c-Raf-RBD(RKY) using a bio-layer interferometry (BLI) assay and found that this new variant exhibits single-digit nanomolar affinity for KRasGTP, confirming the computational predictions made with O_SCPLOWFRIESC_SCPLOW/EWAK*. This study steps through the advancement and development of computational protein design by presenting theory, new algorithms, accurate retrospective designs, new prospective designs, and biochemical validation.\n\nAuthor summaryComputational structure-based protein design is an innovative tool for redesigning proteins to introduce a particular or novel function. One such possible function is improving the binding of one protein to another, which can increase our understanding of biomedically important protein systems toward the improvement or development of novel therapeutics. Herein we introduce two novel, provable algorithms, O_SCPLOWFRIESC_SCPLOW and EWAK*, for more efficient computational structure-based protein design as well as their application to the redesign of the c-Raf-RBD:KRas protein-protein interface. These new algorithms speed up computational structure-based protein design while maintaining accurate calculations, allowing for larger, previously infeasible protein designs. Using O_SCPLOWFRIESC_SCPLOW and EWAK* within the O_SCPLOWOSPREYC_SCPLOW suite, we designed the tightest known binder of KRas, an \"undruggable\" cancer target. This new variant of a KRas-binding domain, c-Raf-RBD, should serve as an important tool to probe the protein-protein interface between KRas and its effectors as work continues toward an effective therapeutic targeting KRas.

biochemistry↗