Research Overview

RNA interference (RNAi) is a widely conserved and naturally occurring process in which small RNAs lead to targeted gene silencing.  In the short time since its discovery in the round worm Caenorhabditis elegans, RNAi has revolutionized our understanding of gene regulation and has been found to play key roles in viral defense mechanisms and in tumor suppression.  Furthermore, researchers are actively developing RNAi-based drugs to silence disease-causing genes in patients.  Some of these drugs are already in Phase II clinical trials.  This new class of drugs has great potential for the treatment of childhood cancers including Ewing’s sarcoma and acute lymphoblastic leukemia, which are associated with specific cancer-causing genes that can be targeted by RNAi.  The biggest challenge facing the effective use of RNAi-based therapies is safe delivery of these drugs to their proper targets while avoiding off-target effects.  RNAi delivery is very efficient in C. elegans, and an important RNA transport protein discovered in the worm has been shown to be important for delivery of guide RNAs in humans.  Our goal is to use C. elegans to identify and characterize additional proteins important for the delivery of RNAi.  This work will allow for subsequent detailed analyses of corresponding human genes and will provide important insights for the delivery of RNAi-based therapies.

About Jennifer Whangbo, MD, PhD

Dr. Whangbo received her Bachelor's in Science from the University of North Carolina, Chapel Hill in 1994. She received her PhD from the University of California, San Francisco in 1999 in Biochemistry and her MD from University of California, Los Angeles in 2003.

Researcher Services

Researcher Areas

  • Hematology/Oncology

Research Departments

Research Divisions

Researcher Centers

PUBLICATIONS

Publications powered by Harvard Catalyst Profiles

  1. Joyce CE, Saadatpour A, Ruiz-Gutierrez M, Bolukbasi OV, Jiang L, Thomas DD, Young S, Hofmann I, Sieff C, Myers KC, Whangbo J, Libermann TA, Nusbaum C, Yuan GC, Shimamura A, Novina CD. TGFß signaling underlies hematopoietic dysfunction and bone marrow failure in Shwachman-Diamond Syndrome. J Clin Invest. 2019 Jun 18; 130. View abstract
  2. Whangbo JS, Kim HT, Nikiforow S, Koreth J, Alho AC, Falahee B, Kim S, Dusenbury K, Fields MJ, Reynolds CG, Alyea EP, Armand P, Cutler CS, Ho VT, Antin JH, Soiffer RJ, Ritz J. Functional analysis of clinical response to low-dose IL-2 in patients with refractory chronic graft-versus-host disease. Blood Adv. 2019 Apr 09; 3(7):984-994. View abstract
  3. Belizaire R, Kim HT, Poryanda SJ, Mirkovic NV, Hipolito E, Savage WJ, Reynolds CG, Fields MJ, Whangbo J, Kubo T, Nikiforow S, Alyea EP, Armand P, Cutler CS, Ho VT, Blazar BR, Antin JH, Ritz J, Soiffer RJ, Koreth J. Efficacy and immunologic effects of extracorporeal photopheresis plus interleukin-2 in chronic graft-versus-host disease. Blood Adv. 2019 Apr 09; 3(7):969-979. View abstract
  4. Alborghetti MR, Correa MEP, Whangbo J, Shi X, Aricetti JA, da Silva AA, Miranda ECM, Sforca ML, Caldana C, Gerszten RE, Ritz J, Zeri ACM. Clinical Metabolomics Identifies Blood Serum Branched Chain Amino Acids as Potential Predictive Biomarkers for Chronic Graft vs. Host Disease. Front Oncol. 2019; 9:141. View abstract
  5. Ben Farhat K, Alosaimi MF, Shendi H, Al-Hammadi S, Jones J, Schwarz K, Schulz A, Alawdah LS, Burchett S, Albuhairi S, Whangbo J, Kwatra N, Shamseldin HE, Alkuraya FS, Chou J, Geha RS. Immunologic reconstitution following hematopoietic stem cell transplantation despite lymph node paucity in NF-?B-inducing kinase deficiency. J Allergy Clin Immunol. 2019 Mar; 143(3):1240-1243.e4. View abstract
  6. Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY. B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation. Blood. 2018 06 28; 131(26):2967-2977. View abstract
  7. Andermann TM, Peled JU, Ho C, Reddy P, Riches M, Storb R, Teshima T, van den Brink MRM, Alousi A, Balderman S, Chiusolo P, Clark WB, Holler E, Howard A, Kean LS, Koh AY, McCarthy PL, McCarty JM, Mohty M, Nakamura R, Rezvani K, Segal BH, Shaw BE, Shpall EJ, Sung AD, Weber D, Whangbo J, Wingard JR, Wood WA, Perales MA, Jenq RR, Bhatt AS. The Microbiome and Hematopoietic Cell Transplantation: Past, Present, and Future. Biol Blood Marrow Transplant. 2018 07; 24(7):1322-1340. View abstract
  8. Whangbo JS, Weisman AS, Chae J, Hunter CP. SID-1 Domains Important for dsRNA Import in Caenorhabditis elegans. G3 (Bethesda). 2017 12 04; 7(12):3887-3899. View abstract
  9. Platt CD, Fried AJ, Hoyos-Bachiloglu R, Usmani GN, Schmidt B, Whangbo J, Chiarle R, Chou J, Geha RS. Combined immunodeficiency with EBV positive B cell lymphoma and epidermodysplasia verruciformis due to a novel homozygous mutation in RASGRP1. Clin Immunol. 2017 10; 183:142-144. View abstract
  10. Hirakawa M, Matos TR, Liu H, Koreth J, Kim HT, Paul NE, Murase K, Whangbo J, Alho AC, Nikiforow S, Cutler C, Ho VT, Armand P, Alyea EP, Antin JH, Blazar BR, Lacerda JF, Soiffer RJ, Ritz J. Low-dose IL-2 selectively activates subsets of CD4+ Tregs and NK cells. JCI Insight. 2016 11 03; 1(18):e89278. View abstract
  11. Whangbo J, Ritz J, Bhatt A. Antibiotic-mediated modification of the intestinal microbiome in allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2017 Feb; 52(2):183-190. View abstract
  12. Koreth J, Kim HT, Jones KT, Lange PB, Reynolds CG, Chammas MJ, Dusenbury K, Whangbo J, Nikiforow S, Alyea EP, Armand P, Cutler CS, Ho VT, Chen YB, Avigan D, Blazar BR, Antin JH, Ritz J, Soiffer RJ. Efficacy, durability, and response predictors of low-dose interleukin-2 therapy for chronic graft-versus-host disease. Blood. 2016 07 07; 128(1):130-7. View abstract
  13. Alho AC, Kim HT, Chammas MJ, Reynolds CG, Matos TR, Forcade E, Whangbo J, Nikiforow S, Cutler CS, Koreth J, Ho VT, Armand P, Antin JH, Alyea EP, Lacerda JF, Soiffer RJ, Ritz J. Unbalanced recovery of regulatory and effector T cells after allogeneic stem cell transplantation contributes to chronic GVHD. Blood. 2016 Feb 04; 127(5):646-57. View abstract
  14. Thomas MP, Liu X, Whangbo J, McCrossan G, Sanborn KB, Basar E, Walch M, Lieberman J. Apoptosis Triggers Specific, Rapid, and Global mRNA Decay with 3' Uridylated Intermediates Degraded by DIS3L2. Cell Rep. 2015 May 19; 11(7):1079-89. View abstract
  15. Thomas MP, Whangbo J, McCrossan G, Deutsch AJ, Martinod K, Walch M, Lieberman J. Leukocyte protease binding to nucleic acids promotes nuclear localization and cleavage of nucleic acid binding proteins. J Immunol. 2014 Jun 01; 192(11):5390-7. View abstract
  16. Whangbo JS, Hunter CP. Environmental RNA interference. Trends Genet. 2008 Jun; 24(6):297-305. View abstract
  17. Smith R, Owen LA, Trem DJ, Wong JS, Whangbo JS, Golub TR, Lessnick SL. Expression profiling of EWS/FLI identifies NKX2.2 as a critical target gene in Ewing's sarcoma. Cancer Cell. 2006 May; 9(5):405-16. View abstract
  18. Ch'ng Q, Williams L, Lie YS, Sym M, Whangbo J, Kenyon C. Identification of genes that regulate a left-right asymmetric neuronal migration in Caenorhabditis elegans. Genetics. 2003 Aug; 164(4):1355-67. View abstract
  19. Whangbo J, Harris J, Kenyon C. Multiple levels of regulation specify the polarity of an asymmetric cell division in C. elegans. Development. 2000 Nov; 127(21):4587-98. View abstract
  20. Whangbo J, Kenyon C. A Wnt signaling system that specifies two patterns of cell migration in C. elegans. Mol Cell. 1999 Nov; 4(5):851-8. View abstract
  21. Maloof JN, Whangbo J, Harris JM, Jongeward GD, Kenyon C. A Wnt signaling pathway controls hox gene expression and neuroblast migration in C. elegans. Development. 1999 Jan; 126(1):37-49. View abstract