About RNA Therapeutics & Biology at UMass Chan

In addition to protein-coding mRNAs, genomes produce a wide variety of non-coding RNAs, which function in a vast array of cellular processes, including all aspects of gene expression and its regulation. The study of RNA Biology focuses on understanding the mechanisms of RNA interference, a form of sequence-specific gene silencing triggered by double-stranded RNA, functions of microRNAs that control timing of organism development, RNA splicing mechanisms and noncoding RNA actions, mRNA translational control, and the role of piRNAs in maintenance of germline integrity. Recent discoveries on the role of RNA in gene editing, including mechanisms of CRISPR, are also proceeding at a rapid pace. RNA biologists across campus have made striking discoveries in novel types of RNA and their mechanisms of action, are investigating the role of RNAs in a variety of human diseases such as cancers, diabetes, autism and Fragile X, and are using these discoveries to fuel the development of RNA therapeutics to treat disease.

Ambros Lab
We study gene regulatory mechanisms controlling the timing of animal development, using the C. elegans model system. Developmental timing regulators in C. elegans include microRNAs that control the stage-specific expression of key transcription factors. We aim to understand the molecular mechanisms of post-transcriptional gene regulation by microRNAs, and how microRNAs function in regulatory networks affecting development and disease. (Ambros profile)

Czech Lab
Our laboratory develops nanoparticles composed of siRNA or CRISPR components for gene silencing and editing in order to discover novel cell signaling pathways related to cancer, diabetes and obesity. We are also interested in advancing these RNA technologies towards therapeutics for these diseases. (Czech profile)

Davis Lab
The cJun NH2-terminal kinase (JNK) signal transduction pathway is implicated in several stress-related disease processes including cancer, diabetes, inflammation, and stroke. Our hope is that drugs targeting the JNK pathway may be useful for the treatment of these diseases. The goal of this laboratory is to understand the molecular processes that are engaged by JNK in both health and disease.  (Davis profile)

Garber Lab
Dr. Garber’s methods have been critical to the discovery and characterization of a novel set of large intergenic non-coding RNAs (lincRNAs) and to our understanding of the immune transcriptional response to pathogens. (Garber profile)

Gregory Lab
Our research is focused on identifying and characterizing new mechanisms of RNA regulation in the dynamic control of gene expression. We apply this knowledge to explore how RNA regulatory pathways impact stem cell pluripotency, mammalian development, growth, cancer, and neurological diseases. Ultimately, we aim to exploit this understanding for the development of new therapeutic approaches for cancer and degenerative disease.

Khvorova Lab
We are a chemical biology lab dedicated to developing RNA-based therapeutics. Our progress stems from asking the right research questions—those rooted in a deep understanding of the biochemical and pharmacological pathways that define and limit the potential of RNA as a drug. Our core mission is to unite informaticians, chemists, biochemists, cell biologists, and pharmacologists in collaborative research. This cross-disciplinary approach has enabled transformative advances in RNA therapeutics—from siRNA informatics and insights into RISC (RNA-induced silencing complex) biology to the development of pharmacological principles and two new classes of therapeutic siRNAs. (Khvorova profile)

Mello Lab
Our lab uses the nematode worm C. elegans as a model organism to investigate how embryonic cells differentiate and communicate during development. In addition, we are investigating the mechanism of RNA interference, a form of sequence-specific gene silencing triggered by double-stranded RNA.  (Mello profile)

Richter Lab
Our lab studies the biochemistry of post-transcriptional gene expression, particularly cytoplasmic polyadenylation and translational control. We also examine how these processes influence early animal development, cell division and cellular senescence, and neuronal synaptic plasticity and memory consolidation.  (Richter profile)

Sontheimer Lab
The overarching, longstanding aim of the Sontheimer Laboratory is to uncover and understand the roles of RNA molecules during gene expression. Our current emphasis is on mechanisms of genetic interference pathways specified by CRISPR loci in pathogenic bacteria. Most notably, we demonstrated that the molecular target of CRISPR interference is DNA rather than RNA. We have also identified unique features of the CRISPR/Cas pathway in meningococcus and demonstrated the suitability of this system as a platform for genome editing in human pluripotent stem cells. We are poised to develop CRISPR-Cas systems to accelerate biomedical research and treat human disease. (Sontheimer profile)

Theurkauf Lab
Work in the lab addresses RNA localization and embryonic patterning, the response of mitotic cells to DNA damage, and small RNA function in germline development. Studies combine high resolution imaging, genetic, and molecular approaches in Drosophila and mammalian cultured cell systems. (Theurkauf profile)

Zamore lab 
Our lab studies small RNA silencing pathways in eukaryotes and prokaryotes, including RNA interference (RNAi), microRNA, and PIWI-interacting RNA pathways. We seek to use these insights to design therapies for human diseases, including Huntington’s disease. Under Dr. Zamore’s mentorship, the Zamore Lab has produced dozens of researchers working at top institutions both in the United States and abroad.