A brunette man with a beard smiling

Maximilian Horlbeck, MD, PhD

Instructor in Pediatrics, Boston Children's Hospital
Horlbeck Lab

The Horlbeck lab studies Chromatinopathies, a large class of genetic disorders in which disruption of chromatin modifying machinery leads to developmental delay and intellectual disability. The lab seeks to understand how chromatin regulation shapes cell fate decisions and identify paths to treatment for these disorders. Approaches include high-throughput genetic screening, genetic interaction mapping, and single cell epigenomics in iPSC-derived models. They also aim to develop new approaches to address the genetic phenomena of incomplete penetrance and variable expressivity that characterize these and other human genetic disorders.

Combinatorial genetics in liver repopulation and carcinogenesis with a in vivo CRISPR activation platform.
Authors: Authors: Wangensteen KJ, Wang YJ, Dou Z, Wang AW, Mosleh-Shirazi E, Horlbeck MA, Gilbert LA, Weissman JS, Berger SL, Kaestner KH.
Hepatology
View full abstract on Pubmed
Promoter of lncRNA Gene PVT1 Is a Tumor-Suppressor DNA Boundary Element.
Authors: Authors: Cho SW, Xu J, Sun R, Mumbach MR, Carter AC, Chen YG, Yost KE, Kim J, He J, Nevins SA, Chin SF, Caldas C, Liu SJ, Horlbeck MA, Lim DA, Weissman JS, Curtis C, Chang HY.
Cell
View full abstract on Pubmed
Identification of a transporter complex responsible for the cytosolic entry of nitrogen-containing bisphosphonates.
Authors: Authors: Yu Z, Surface LE, Park CY, Horlbeck MA, Wyant GA, Abu-Remaileh M, Peterson TR, Sabatini DM, Weissman JS, O'Shea EK.
Elife
View full abstract on Pubmed
Targeting RAS-driven human cancer cells with antibodies to upregulated and essential cell-surface proteins.
Authors: Authors: Martinko AJ, Truillet C, Julien O, Diaz JE, Horlbeck MA, Whiteley G, Blonder J, Weissman JS, Bandyopadhyay S, Evans MJ, Wells JA.
Elife
View full abstract on Pubmed
Combined CRISPRi/a-Based Chemical Genetic Screens Reveal that Rigosertib Is a Microtubule-Destabilizing Agent.
Authors: Authors: Jost M, Chen Y, Gilbert LA, Horlbeck MA, Krenning L, Menchon G, Rai A, Cho MY, Stern JJ, Prota AE, Kampmann M, Akhmanova A, Steinmetz MO, Tanenbaum ME, Weissman JS.
Mol Cell
View full abstract on Pubmed
CRISPRi-based genome-scale identification of functional long noncoding RNA loci in human cells.
Authors: Authors: Liu SJ, Horlbeck MA, Cho SW, Birk HS, Malatesta M, He D, Attenello FJ, Villalta JE, Cho MY, Chen Y, Mandegar MA, Olvera MP, Gilbert LA, Conklin BR, Chang HY, Weissman JS, Lim DA.
Science
View full abstract on Pubmed
A Multiplexed Single-Cell CRISPR Screening Platform Enables Systematic Dissection of the Unfolded Protein Response.
Authors: Authors: Adamson B, Norman TM, Jost M, Cho MY, Nuñez JK, Chen Y, Villalta JE, Gilbert LA, Horlbeck MA, Hein MY, Pak RA, Gray AN, Gross CA, Dixit A, Parnas O, Regev A, Weissman JS.
Cell
View full abstract on Pubmed
Compact and highly active next-generation libraries for CRISPR-mediated gene repression and activation.
Authors: Authors: Horlbeck MA, Gilbert LA, Villalta JE, Adamson B, Pak RA, Chen Y, Fields AP, Park CY, Corn JE, Kampmann M, Weissman JS.
Elife
View full abstract on Pubmed
Versatile in vivo regulation of tumor phenotypes by dCas9-mediated transcriptional perturbation.
Authors: Authors: Braun CJ, Bruno PM, Horlbeck MA, Gilbert LA, Weissman JS, Hemann MT.
Proc Natl Acad Sci U S A
View full abstract on Pubmed
Parallel shRNA and CRISPR-Cas9 screens enable antiviral drug target identification.
Authors: Authors: Deans RM, Morgens DW, Ökesli A, Pillay S, Horlbeck MA, Kampmann M, Gilbert LA, Li A, Mateo R, Smith M, Glenn JS, Carette JE, Khosla C, Bassik MC.
Nat Chem Biol
View full abstract on Pubmed