Syllabus
Fall, 2012
Topics in Molecular Medicine
Wednesdays (unless noted) from 3:30 pm - 5:00 pm in room 601 BRB
Class #1 – Robert Doms – August 29 - "The Berlin patient and a cure for HIV/AIDS"
- Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM (2010):Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 28(8):839-47. [pdf]
- Hutter G, Nowak D, Mossner M, Ganepola S, Mussig A, Allers K, Schneider T, Hofmann J, Kucherer C, Blau O, Blau IW, Hofmann Wk, Thiel E (2009): Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. NEJM 360(7):692-8. [pdf]
- Background Review
Class #2 – Bob Vonderheide – September 5 -"Improving Cancer Vaccines"
- Kraman M, Bambrough PJ, Arnold JN, Roberts EW, Magiera L, Jones JO, Gopinathan A, Tuveson DA, Fearon DT (2010): Suppression of antitumor immunity by stromal cells expressing fibroblast activation protein-alpha. Science 330(6005):827-30. [pdf]
- Finn OJ (2008): Cancer Immunology. NEJM 358(25):2704-15. [pdf]
Class #3 – Nancy Spinner & Ian Krantz – September 12 - "Prenatal genomics: Amazing advances and diagnostic dilemmas"
- Kitzman JO, Snyder MW, Ventura M, Lewis AP, Qiu R, Simmons LE, Gammill HS, Rubens CE, Santillan DA, Murray JC, Tabor HK, Bamshad MJ, Eichler EE, Shendure J (2012). Noninvasive whole-genome sequencing of a human fetus. Sci Transl Med. 4(137):137ra76. [pdf]
- Arnold KMR and Self ZB (2012) Genetic Screening and Counseling: Family Medicine Obstetrics Prim Care Clin Office Pract 39:55-70. [pdf]
- Supplemental paper for discussion
- Benn PA and Chapman AR (2010). Ethical Challenges in providing noninvasive prenatal diagnosis. Current Opinion in Obstetrics and Gynecology 22:128-134. [pdf]
Class #4 – Joshua Lipshutz – September 19 - "Autosomal dominant polycystic kidney disease: From bench to bedside and back"
- Gattone VH 2nd, Wang X, Harris PC, Torres VE (2003). Inhibition of renal cystic disease development and progression by a vasopressin V2 receptor antagonist. Nature Medicine 9(10):1323-6. [pdf]
- Torres VE, Harris PC, Pirson Y (2007). Autosomal dominant polycystic kidney disease. The Lancet 369: 1287-301. [pdf]
Class #5 – Kathy High – September 24 (Monday) - "Gene therapy for Thalassemia"
- Cunningham MJ, Weis MJ, Neufeld EJ. Thalassemia. In: Clinical Hematology, 1st Ed, Young NS, Gerson SL and High KA (eds.), Elsevier Science, Philadelphia, PA, chapter 21, pp. 281-292, 2006. [pdf]
- Cavazzana-Calvo M, Payen E, Negre O, et al (2010). Transfusion Independence and HMGA2 Activation after Gene Therapy of Human Beta-Thalasesemia. Nature 467:318-322. [pdf]
Class #6 – Andy Minn – October 3 –" Cancer genomics and the goal of personalized medicine"
- Gerlinger M, et. al. (2012). Intratumor Heterogeneity and Branched Evolution Revealed by Multiregion Sequencing. NEJM 366(10): 883-92. [pdf]
- Navin N, Kendall J, Troge J, Andrews P, Rodgers L, McIndoo J, et. al. (2011). Tumor evolution inferred by single-cell sequencing. Nature 472(7341):90-4. [pdf]
Class #7 – Joshua Friedman – October 17 (Monday) - "The intestinal microbiome and GVHD"
- Jeng RR, Ubeda C, Taur Y, Menezes CC, Khanin R, Dudakov JA, Liu C, West ML, Singer NV, Equinda MJ, Gobourne A, Lipuma L, Young LF, Smith OM, Ghosh A, Hanash AM, Goldberg JD, Aoyama K, Blazar BR, Pamer EG, van den Brink MR (2012). Regulation of intestinal inflammation by microbiota following allogeneic bone marrow transplantation. J Exp Med 209(5): 903-11. [pdf]
- Hooper LV, Littman DR, Macpherson AJ (2012). Interactions Between the Microbiota and the Immune System. Science 336: 1268-73. [pdf]
- Bolaños-Meade J, Vogelsang GB (2004): Acute graft-versus-host disease. Clin Adv Hematol Oncol. 2(10): 672-82. [pdf]
Class #8 – Geoff Aguirre – October 24 - "Neuroimaging to guide deep brain stimulation for treatment of depression"
- Ressler KJ, Mayberg HS (2007). Targeting abnormal neural circuits in mood and anxiety disorders: from the laboratory to the clinic. Nat Neurosci. 10(9):1116-24. [pdf]
- Sheline YI, Price JL, Yan Z, Mintun MA (2010). Resting-state functional MRI in depression unmasks increased connectivity between networks via the dorsal nexus. PNAS 107(24):11020-5. [pdf]
Class #9 – Mark Kahn – October 31 - "Vascular regulation of organ regeneration and growth”
- Kajstura J, Rota M, Hall SR, Hosoda T, D'Amario D, et. al. (2011). Evidence for Human Lung Stem Cells. N Engl J Med 364(19):1795-806. [pdf]
- Ding BS, Noan DJ, Guo P, Babazadeh AO, Cao Z, Rosenwaks Z, Crystal RG, Simons M, Sato TN, Worgall S, Shido K, Rabbany SY, Rafii S (2011). Endothelial-derived angiocrine signals induce and sustain regenerative lung alveolarization. Cell 147(3):539-53. [pdf]
- Supplemental Papers
- Ding BS, Nolan DJ, Butler JM, James D, Babazadeh AO, Rosenwaks Z, Mittal V, Kobayashi H, Shido K, Lyden D, Sato TN, Rabbany SY, Rafii S (2010). Inductive angiocrine signals from sinusoidal endothelium are required for liver regeneration. Nature 468(7321): 310-5. [pdf]
- Chapman HA (2011). Toward lung regeneration. N Engl J Med 364(19): 1867-8. [pdf]
Class #10 – Rahul Kohli – November 7 - “Broadly neutralizing antibody response to HIV”
- Johnston MI, Fauci AS (2011). HIV vaccine development-Improving on natural immunity. N Engl J Med 365(10): 873-5. [pdf]
- Wu X, Zhou T, Zhu J, Zhang B, Georgiev I, Wang C, Chen X, et. al. (2011). Focused evolution of HIV-1 neutralizing antibodies revealed by structures and deep sequencing. Science 333(6049):1593-602. [pdf]
Class #11 – Gerd Blobel – November 14 - "Targeting chromatin readers to treat malignancies"
- Delmore JE, Issa GC, Lemieux ME, Rahl PB, Shi J, Jacobs HM, et. al. (2011). BET Bromodomain inhibition as a therapeutic strategy to target c-Myc. Cell 146(6):904-17. [pdf]
- Palumbo A and Anderson K (2011). Multiple Myeloma. N Engl J Med 364(11):1046-60. [pdf]
Class #12 – Brad Johnson – November 26 (Monday) - "Cellular senescence and age-related pathology”
- Merideth MA, Gordon LB, Clauss S, Sachdev V, Smith AC, Perry MB, Brewer CC, Zalewski C, Kim HJ, Solomon B, et. al. (2008): Phenotype and course of Hutchinson-Gilford progeria syndrome. NEJM 358(6):592-604. [pdf]
- Baker DF, Wijshake T, Tchkonia T, LeBrasseur NK, Childs BG, van de Sluis B, Kirkland JL, van Deursen JM (2011). Clearance of p16Ink4a-positive senescent cells delays ageing-associated disorders. Nature 479(7372):232-6. [pdf]
- Optional review: Campisi J, Andersen JK, Kapahi P, Melov S (2011). Cellular senescence: A link between cancer and age-related degenerative disease? Semin Cancer Biol 21(6):354-9. [pdf]
Class #13 – Monica Bessler – November 28 - "The role of p53/p21 in mechanisms of bone marrow failure in patients with Fanconi Anemia"
- Soulier J (2011). Fanconi Anemia. Hematology Am Soc Hematol Educ Program 2011:492-7. [pdf]
- Ceccaldi R, Parmar K, Mouly E, Delord M, Kim JM, Regairaz M, Pla M, Vasquez N, Zhang QS, et. al. (2012): Bone Marrow Failure in Fanconi Anemia is Triggered by an Exacerbated p53/p21 DNA Damage Response that Impairs Hematopoietic Stem and Progenitor Cells. Cell Stem Cell 11(1):36-49. [pdf]
- Supplemental data [pdf]
Class #14 – Aimee Payne – December 3 (Monday) - "Beyond gene therapy: Strategies for correcting epidermolysis bullosa"
- M. Peter Marinkovich, Paul A. Khavari, G. Scott Herron and Eugene A. Bauer: Inherited Epidermolysis Bullosa. Chapter 65, DIGM Review. 596-609. [pdf]
- Woodley DT, Keene DR, Atha T, Huang Y, Lipman K, Li W, Chen M (2004). Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa. Nat Med 10(7):693-5. [pdf]
Class #15 – Dorris Stoffers – December 10 (Monday) - "Cell-based therpies for Type 1 Diabetes"
- Powers AC. Chapter 344: Diabetes Mellitus. In: Longo DL, Fauci AS, Kasper DL, Hauser SL, Jameson JL, Loscalzo J, eds. Harrison's Principles of Internal Medicine. 18th ed. New York: McGraw-Hill; 2012. [pdf]
- Zhou Z, Brown J, Kanarek A, Rajagopal J, Melton DA (2008). In vivo reprogramming of adult pancreatic exocrine cells to beta-cells. Nature 455(7213):627-32. [pdf]
• Last updated: 11/13/2012