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education and training: Hardin-Simmons University, Biology
education and training: University of Houston, Biotechnology
education and training: University of Massachusetts Medical School, Biomedical Sciences
research areas: Gene Expression Regulation, Developmental
research areas: Caenorhabditis elegans
research areas: Oligonucleotides, Antisense
research areas: Endothelial Cells
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linked information resource: Effects of Larval Density on Gene Regulation in Caenorhabditis elegans During Routine L1 Synchronization.

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linked information resource: Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection.

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linked information resource: Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts.

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linked information resource: A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors.

Caenorhabditis elegans
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Xue, Wen
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Post Docs: We are looking for postdoc candidates with experience in cancer biology, mouse models and genome-engineering. Please email CV and 3 letters of recommendation to Wen.Xue@umassmed.edu.
Rotation Projects: Our lab uses CRISPR tools to speed up cancer gene discovery and disease gene repair. Rotation projects are available to explore experimental techniques such as molecular cloning of CRISPR plasmids, measuring genome-editing efficiency, dissecting mice, and processing of mouse tumor/tissue for histology.
preferred title: Professor
webpage: https://profiles.umassmed.edu/profile/28866638

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Mello, Craig
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Rotation Projects: Potential Rotation Projects: There are numerous potential rotation project available in the Mello lab: Projects include: Investigation of RNAi and related mechanisms, including Piwi Argonaute pathways and their affects on fertility and epigenetic inheritance; Investigation of germline determination and cell-fate specification during embryogenesis; the exploration of CRISPR mediated genome editing tools for C. elegans and other organisms; and finally the use of CRISPR to generate C. elegans models for human genetic disease.
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Rando, Oliver
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Endowed Chair Honorary Title: Endowed Chair in Biochemistry and Molecular Biotechnology II
Post Docs: For more information about the Postdoctoral position in Dr. Rando’s lab please use the link provided. Post doc position
Rotation Projects: Rotation projects are available to study chromatin structure and function. Project 1. Measure histone dynamics in yeast. Histone dynamics play major roles in chromatin metabolism and in the inheritance of chromatin states. To measure histone replacement rates in yeast, we have created yeast strains carrying inducible epitope-tagged histones H3 and H2A. Using microarrays, we will measure histone replacement rates in a variety of mutant yeast to ascertain the role of various chromatin remodeling factors in histone exchange reactions. Project 2. Characterize mechanisms for epigenetic inheritance in yeast. We have identified a variety of genes whose expression state in yeast is epigenetically heritable. We will screen the yeast genome for genes that affect the inheritance of these expression patterns. Project 3. Measure histone dynamics in embryonic stem cells. Embryonic stem cells are pluripotent cells that can give rise to every type of cell in the adult animal. The role of chromatin structure in maintaining this pluripotent state is poorly understood, but it appears that stem cell chromatin is exceptionally labile relative to that in differentiated cells. We will create stem cell lines with inducible tagged histones to measure histone replacement rates across the mouse genome.
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Ambros, Victor
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Rotation Projects: Rotations <p >Project 1: Genetic screens for proteins that modify or regulate the activity of microRNAs in C. elegans.
preferred title: Professor

Gao, Guangping
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Person ID: 1177
person in primary position: Chair and Professor
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Endowed Chair Honorary Title: Penelope Booth Rockwell Chair in Biomedical Research
Lab URL: https://www.umassmed.edu/gaolab/
Summary: Focus: Adeno-associated virus vector mediated gene transfer for gene therapy, gene therapy of inherited neurodegenerative Canavan Disease
preferred title: Chair and Professor

Khvorova, Anastasia
first name: Anastasia
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Person ID: 3413
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Endowed Chair Honorary Title: Remondi Family Chair in Biomedical Research
Post Docs: POSTDOCTORAL POSITION: RNAI PRECLINICAL DEVELOPMENT Excellent training opportunity for a person considering industrial carrier in drug development Oligonucleotides represent a new class of drugs, which have the promise to become a major class of future therapeutics. There are several classes of oligonucleotides which can efficiently silence genes in various tissues with acceptable level of toxicity. There are also new chemistries being developed to further improve oligonucleotide tissue distribution and cellular uptake. The successful candidate will be a key part of an intra-disciplinary team working with biologists and chemists to develop novel types of therapeutics for treatment of neurological disease, liver disease and orphan indications. The work will include compounds identification, screening, characterization, in vivo efficacy and PK/PD studies, novel assays development etc. In addition exposure and training in nucleic acid pre-clinical development, project management etc will be provided. QUALIFICATIONS: Applicants must have (or expect to obtain shortly) a Ph.D. in Chemistry, Organic Chemistry Biochemistry, Cell Biology, Molecular Biology or a related field , be bright, ambitious, independent and collaborative and ideally, have research experience in nucleic acid molecular cell biology or pharmacology.
Post Docs: RNAI MEDICINAL CHEMISTRY/ RNA OLIGONUCLEOTIDE CHEMISTRY Develop and characterize novel RNA chemistries to promote efficient oligonucleotide internalization and tissue distribution. Oligonucleotides represent a new class of drugs, which have the promise to become a major class of future therapeutics. There are several chemistries and formulations developed by us and others that effectively support oligo internalization and favorable PK/PD. In most cases in vivo efficacy is limited by (1) poor PK (2) inefficient tissue distribution (3) compound entrapment in biologically inactive intracellular compartments. The objective of future studies will be development of novel chemistries, which promote receptor mediated cellular internalization and/ or endosomal escape. The successful candidate will be responsible for the design and synthesis of novel RNA precursors, optimization of RNA synthesis and conjugation protocols and interface with the biology part of the lab. In addition, exposure to pre-clinical development programs and oligonucleotide manufacturing will be available. QUALIFICATIONS: Applicants must have (or expect to obtain shortly) a Ph.D. in Chemistry, Organic Chemistry Biochemistry, Cell Biology, Molecular Biology or a related field , be bright, ambitious, independent and collaborative and have previous experience in nucleic acid / oligonucleotide chemistry.
Post Docs: SMALL RNA TRAFFICKING Discover, characterize and adapt mechanisms of small RNA extracellular and intracellular trafficking for development of novel classes of oligonucleotide drugs. A serious limitation in realizing the potential of oligonucleotide based therapies has been the exceedingly inefficient transit of oligonucleotides from outside cells to the intracellular compartments where the biologically relevant activity happens. On the other hand, there are multiple lines of evidence supporting the notion that nature has implemented evolutionarily conserved mechanisms and pathways for efficiently trafficking small RNAs across cellular boundaries. The objective of future studies will be the systematic evaluation of mechanisms of cellular uptake of different classes of oligonucleotides and their comparison to mechanisms of native RNA trafficking using a combination of experimental approaches, including advanced RNA chemistry and high temporal and spatial resolution microscopy in living cells, Mass spectrometry, cell biology and others. The successful candidate will work closely with Victor Ambros and Silvia Corvera labs and the UMASS imaging group and receive training in RNA chemistry, biochemistry, cell biology and pre-clinical development. QUALIFICATIONS:Applicants must have (or expect to obtain shortly) a Ph.D. in Chemistry, Organic Chemistry Biochemistry, Cell Biology, Molecular Biology or a related field , be bright, ambitious, independent and collaborative and ideally, have research experience in nucleic acid molecular and cell biology
Rotation Projects: Our laboratory uses a combination of nucleic acid and oligonucleotide chemistry, biochemistry, cell biology, and pharmacology and develops and evaluate novel technologies for development of oligonucleotide therapeutics. A rotation project is available to develop and identify chemically modified self-delivering RNAi compounds for treatment of Huntington and other diseases.
preferred title: Professor

Watts, Jonathan
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Hardin-Simmons University, Biology
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University of Houston, Biotechnology
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University of Massachusetts Medical School, Biomedical Sciences
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Effects of Larval Density on Gene Regulation in Caenorhabditis elegans During Routine L1 Synchronization.
PubMed Identifier: 29602810
author list: Chan IL, Rando OJ, Conine CC
published in: G3 (Bethesda, Md.)
information resource reference: Chan IL, Rando OJ, Conine CC. Effects of Larval Density on Gene Regulation in Caenorhabditis elegans During Routine L1 Synchronization. G3 (Bethesda). 2018 05 04; 8(5):1787-1793.
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Biochemistry and Molecular Biotechnology
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Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts.
PubMed Identifier: 35920332
author list: Shin M, Chan IL, Cao Y, Gruntman AM, Lee J, Sousa J, Rodr?guez TC, Echeverria D, Devi G, Debacker AJ, Moazami MP, Krishnamurthy PM, Rembetsy-Brown JM, Kelly K, Yukselen O, Donnard E, Parsons TJ, Khvorova A, Sontheimer EJ, Maehr R, Garber M, Watts JK
published in: Nucleic acids research
information resource reference: Shin M, Chan IL, Cao Y, Gruntman AM, Lee J, Sousa J, Rodr?guez TC, Echeverria D, Devi G, Debacker AJ, Moazami MP, Krishnamurthy PM, Rembetsy-Brown JM, Kelly K, Yukselen O, Donnard E, Parsons TJ, Khvorova A, Sontheimer EJ, Maehr R, Garber M, Watts JK. Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts. Nucleic Acids Res. 2022 08 26; 50(15):8418-8430.
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Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection.
PubMed Identifier: 36893269
author list: Hariharan VN, Shin M, Chang CW, O'Reilly D, Biscans A, Yamada K, Guo Z, Somasundaran M, Tang Q, Monopoli K, Krishnamurthy PM, Devi G, McHugh N, Cooper DA, Echeverria D, Cruz J, Chan IL, Liu P, Lim SY, McConnell J, Singh SP, Hildebrand S, Sousa J, Davis SM, Kennedy Z, Ferguson C, Godinho BMDC, Thillier Y, Caiazzi J, Ly S, Muhuri M, Kelly K, Humphries F, Cousineau A, Parsi KM, Li Q, Wang Y, Maehr R, Gao G, Korkin D, McDougall WM, Finberg RW, Fitzgerald KA, Wang JP, Watts JK, Khvorova A
published in: Proceedings of the National Academy of Sciences of the United States of America
information resource reference: Hariharan VN, Shin M, Chang CW, O'Reilly D, Biscans A, Yamada K, Guo Z, Somasundaran M, Tang Q, Monopoli K, Krishnamurthy PM, Devi G, McHugh N, Cooper DA, Echeverria D, Cruz J, Chan IL, Liu P, Lim SY, McConnell J, Singh SP, Hildebrand S, Sousa J, Davis SM, Kennedy Z, Ferguson C, Godinho BMDC, Thillier Y, Caiazzi J, Ly S, Muhuri M, Kelly K, Humphries F, Cousineau A, Parsi KM, Li Q, Wang Y, Maehr R, Gao G, Korkin D, McDougall WM, Finberg RW, Fitzgerald KA, Wang JP, Watts JK, Khvorova A. Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection. Proc Natl Acad Sci U S A. 2023 03 14; 120(11):e2219523120.
journal title abbreviation: Proc Natl Acad Sci U S A
publication date: 2023-03-09T00:00:00
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A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors.
PubMed Identifier: 38108517
author list: Chen Z, Kwan SY, Mir A, Hazeltine M, Shin M, Liang SQ, Chan IL, Kelly K, Ghanta KS, Gaston N, Cao Y, Xie J, Gao G, Xue W, Sontheimer EJ, Watts JK
published in: The CRISPR journal
information resource reference: Chen Z, Kwan SY, Mir A, Hazeltine M, Shin M, Liang SQ, Chan IL, Kelly K, Ghanta KS, Gaston N, Cao Y, Xie J, Gao G, Xue W, Sontheimer EJ, Watts JK. A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors. CRISPR J. 2023 12; 6(6):570-582.
journal title abbreviation: CRISPR J
publication date: 2023-12-01T00:00:00
year: 2023
type: Academic Article
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Hariharan, Vignesh
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