Publications

Kotterman, M. and D.V. Schaffer (2014) “Engineering Adeno-Associated Viruses for Clinical Gene Therapy.” Nature Reviews Genetics, 15:445-451 doi:10.1038/nrg3742

Dalkara, D., L.C. Byrne, R.R. Klimczak, M. Visel, L. Yin, W.H. Merigan, J.G. Flannery, and D.V. Schaffer (2013) “In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer-Retinal Gene Delivery from the Vitreous.” Science Translational Medicine, 5:189ra76 (PMID 23761039).

Schaffer, D. (2014), “Directed evolution of novel adeno-associated viral vectors for gene therapy”, in Naldini, L. (ed.), Gene Transfer and Gene Therapy: , The Biomedical & Life Sciences Collection, Henry Stewart Talks Ltd, London

Ojala, D., D.P. Amara, and D.V. Schaffer “Adeno-Associated Virus Vectors and Neurological Gene Therapy.” Neuroscientist, Advanced Online Publication: doi:10.1177/1073858414521870

Byrne, L.C., B.E. Öztürk, T. Lee, C. Fortuny, M. Visel, D. Dalkara, D.V. Schaffer, and J.G. Flannery (2014) “Retinoschisin Gene Therapy in Photoreceptors, Müller Glia or All Retinal Cells in the Rs1h-/- Mouse.” Gene Therapy, 21:585-592 (PMID: 24694538).

Vacca, O., M. Darche, D.V. Schaffer, J.G. Flannery, J.A. Sahel, A. Rendon, and D. Dalkara (2014) “AAV-mediated Gene Delivery in Dp71-null Mouse Model with Compromised Barriers.” Glia, 62:468-476 (PMID 24382652).

Byrne, L.C., F. Khalid, T. Lee, E.A. Zin, K.P. Greenberg, M. Visel, D.V. Schaffer, and J.G. Flannery (2013) “AAV-mediated, Optogenetic Ablation of Müller Glia Leads to Structural and Functional Changes in the Mouse Retina.” Public Library of Science (PLoS) ONE, 8:e76075 (PMID 24086689).

Asuri, P., M.A. Bartel, T. Vazin, J-H. Jang, T.B. Wong, and D.V. Schaffer (2012) “Directed Evolution of Adeno-Associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells.” Molecular Therapy, 20:329-38 (PMID 22108859).

Dalkara, D., K.D. Kolstad, K.I. Guerin, N.V. Hoffmann, M. Visel, R.R. Klimczak, D.V. Schaffer, and J.G. Flannery (2011) “AAV-mediated GDNF Secretion from Retinal Glia Slows Down Retinal Degeneration in a Rat Model of Retinitis Pigmentosa.” Molecular Therapy, 19:1602-1608 (PMID 21522134)

Jang, J-H., J.T. Koerber, J-S. Kim, P. Asuri, T. Vazin, M. Bartel, A. Keung, I. Kwon, K.I. Park, and D.V. Schaffer (2011) “An Evolved Adeno-Associated Viral Variant Enhances Gene Delivery and Gene Targeting in Neural Stem Cells.” Molecular Therapy, 19:667-675 (PMID 21224831)

Excoffon, K.J., J.T. Koerber, D.D. Dickey, M. Murtha, S. Keshavjee, B.K. Kaspar, J. Zabner, and D.V. Schaffer (2009) “Directed Evolution of Adeno-Associated Virus to an Infectious Respiratory Virus.” Proceedings of the National Academy of Sciences USA, 106:3865-3870 (PMID 19237554).

Klimczak, R., J.T. Koerber, D. Dalkara, J.G. Flannery, and D.V. Schaffer (2009) “A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller Cells.” Public Library of Science (PLoS) ONE, 4:e7467 (PMID: 19826483).

Koerber, J.T., R. Klimczak, J-H. Jang, D. Dalkara, J.G. Flannery, and D.V. Schaffer (2009) “Molecular Evolution of Adeno-Associated Virus for Enhanced Glial Gene Delivery.” Molecular Therapy, 17: 2088-2095 (PMID: 19672246).

Koerber, J.T., J-H. Jang, and D.V. Schaffer (2008) ” DNA Shuffling of Adeno-Associated Virus Yields Functionally Diverse Viral Progeny.” Molecular Therapy, 16:1703-9 (PMID: 18728640)

Koerber, J.T. and D.V. Schaffer (2008) “Transposon-Based Mutagenesis Generates Diverse Adeno-Associated Viral Libraries with Novel Gene Delivery Properties.” Methods in Molecular Biology, 434:161-170 (PMID 18470644).

Koerber, J.T., J-H. Jang, and D.V. Schaffer (2007) “Engineering Adeno-Associated Virus for One Step Purification via Immobilized Metal Affinity Chromatography.” Human Gene Therapy, 18:367-378 (PMID 17437357).

Koerber, J.T., N. Maheshri, B.K. Kaspar, and D.V. Schaffer (2006) “Construction of Diverse Adeno-Associated Viral Libraries for Directed Evolution of Enhanced Gene Delivery Vehicles.” Nature Protocols, 1:701-706 (PMID 17406299).

Maheshri, N., J.T. Koerber, B. Kaspar, and D.V. Schaffer (2006) “Directed Evolution of Adeno-Associated Virus Yields Enhanced Gene Delivery Vectors.” Nature Biotechnology, 24:198-204 (PMID 16429148).

Maheshri, N. and D.V. Schaffer (2003) “Computational and Experimental Analysis of DNA Shuffling.” Proceedings of the National Academy of Sciences USA, 100:3071-3076 (PMID 12626764).

Randomized dose-finding clinical trial of oncolytic immunotherapeutic vaccinia JX-594 in liver cancer.
Heo J, Reid T, Ruo L, Breitbach CJ, Rose S, Bloomston M, Cho M, Lim HY, Chung HC, Kim CW, Burke J, Lencioni R, Hickman T, Moon A, Lee YS, Kim MK, Daneshmand M, Dubois K, Longpre L, Ngo M, Rooney C, Bell JC, Rhee BG, Patt R, Hwang TH, Kirn DH.
Nature Med. 2013 Mar;19(3):329-36.

Intravenous delivery of a multi-mechanistic cancer-targeted oncolytic poxvirus in humans.
Breitbach CJ, Burke J, Jonker D, Stephenson J, Haas AR, Chow LQ, Nieva J, Hwang TH, Moon A, Patt R, Pelusio A, Le Boeuf F, Burns J, Evgin L, De Silva N, Cvancic S, Robertson T, Je JE, Lee YS, Parato K, Diallo JS, Fenster A, Daneshmand M, Bell JC, Kirn DH.
Nature. 2011 Aug 31;477(7362):99-102.

Targeted and armed oncolytic poxviruses: a novel multi-mechanistic therapeutic class for cancer.
Kirn DH, Thorne SH.
Nature Reviews Cancer. 2009 Jan;9(1):64-71.

Oncolytic and immunotherapeutic vaccinia induces antibody-mediated complement-dependent cancer cell lysis in humans.
Kim MK, Breitbach CJ, Moon A, Heo J, Lee YK, Cho M, Lee JW, Kim SG, Kang DH, Bell JC, Park BH, (KirnDH, Hwang TH co-senior, co-corresponding).
Science Transl Med. 2013 May 15;5(185)

Use of a targeted oncolytic poxvirus, JX-594, in patients with refractory primary or metastatic livercancer: a phase I trial.
Park BH, Hwang T, Liu TC, Sze DY, Kim JS, Kwon HC, Oh SY, Han SY, Yoon JH, Hong SH, Moon A, Speth K, Park C, Ahn YJ, Daneshmand M, Rhee BG, Pinedo HM, Bell JC, Kirn DH.
Lancet Oncol. 2008 Jun;9(6):533-42.

MicroRNAs fine-tune oncolytic viruses.
Bell JC, Kirn D.
Nature Biotechnology. 2008 Dec;26(12):1346-8.

Replication-selective virotherapy for cancer: Biological principles, risk management and future directions.
Kirn D, Martuza RL, Zwiebel J.
Nature Med. 2001 Jul;7(7):781-7.

An adenovirus E1A mutant that demonstrates potent and selective systemic anti-tumoral efficacy.
Heise C, Hermiston T, Johnson L, Brooks G, Sampson-Johannes A, Williams A, Hawkins L, Kirn D.
Nature Med. 2000 Oct;6(10):1134-9.

A controlled trial of intratumoral ONYX-015, a selectively-replicating adenovirus, in combination with cisplatin and 5-fluorouracil in patients with recurrent head and neck cancer.
Khuri FR, Nemunaitis J, Ganly I, Arseneau J, Tannock IF, Romel L, Gore M, Ironside J, MacDougall RH, Heise C, Randlev B, Gillenwater AM, Bruso P, Kaye SB, Hong WK, Kirn DH.
Nature Med. 2000 Aug;6(8):879-85.

ONYX-015, an E1B gene-attenuated adenovirus, causes tumor-specific cytolysis and antitumoral efficacy that can be augmented by standard chemotherapeutic agents.
Heise C, Sampson-Johannes A, Williams A, McCormick F, Von Hoff DD, Kirn DH.
Nature Med. 1997 Jun;3(6):639-45.

Oncolytic vaccinia virus disrupts tumor-associated vasculature in humans.
Breitbach CJ, Arulanandam R, De Silva N, Thorne SH, Patt R, Daneshmand M, Moon A, Ilkow C, Burke J, Hwang TH, Heo J, Cho M, Chen H, Angarita FA, Addison C, McCart JA, Bell JC, Kirn DH.
Cancer Res. 2013 Feb 15;73(4):1265-75.

Targeting of interferon-beta to produce a specific, multi-mechanistic oncolytic vaccinia virus.
Kirn DH, Wang Y, Le Boeuf F, Bell J, Thorne SH.
PLoS Med. 2007 Dec;4(12):e353.

Rational strain selection and engineering creates a broad-spectrum, systemically effective oncolytic poxvirus, JX-963.
Thorne SH, Hwang TH, O’Gorman WE, Bartlett DL, Sei S, Kanji F, Brown C, Werier J, Cho JH, Lee DE, Wang Y, Bell J, Kirn DH.
J Clin Invest. 2007 Nov;117(11):3350-8.

Clinical trial results with oncolytic virotherapy: a century of promise, a decade of progress.
Liu TC, Galanis E, Kirn D.
Nature Reviews Clin Oncol (prev NCPO). 2007 Feb;4(2):101-17.

E3 gene manipulations affect oncolytic adenovirus activity in immunocompetent tumor models.
Wang Y, Hallden G, Hill R, Anand A, Liu TC, Francis J, Brooks G, Lemoine N, Kirn D.
Nature Biotechnol. 2003 Nov;21(11):1328-35.