The following is a partial listing of publications by the Company's two founders, Dr. Matthew J. During and Dr. Michael G. Kaplitt.

1) Kaplitt, M.G., Leone, P., Xiao, X., Samulski, R.J, Pfaff, D.W., O'Malley, K.L. and During, M.J. (1994) Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genetics 8, 148-154.

2) During, M.J., Naegele, J., O'Malley, K.L. and Geller, A.I. (1994) Long-term behavioral recovery in Parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266, 1399-1403.

3) During, M.J., Ryder K.M. and Spencer, D.D. (1995) Hippocampal GABA transporter function in temporal lobe epilepsy. Nature 376, 174-177.

4) Geller, A.I., Naegele, J.R., O'Malley, K.L. and During, M.J. (1995) Behavioral effects and gene delivery in parkinson rat model. Science 269, 856-857.

5) During, M.J. (1996) Gene therapy in New Zealand. Science 272, 467.

6) During, M.J. (1996) Gene Therapy Chronology. Science 273, 416-417.

7) During, M.J., Xu, R., Young, D., Kaplitt, M.G., Sherwin, R.S. and Leone, P. (1998) Peroral gene therapy of lactose intolerance using an AAV vector. Nature Medicine 4, 1131-1135.

8) Young, D., Lawlor, P., Leone, P., Dragunow, M. and During, M.J. (1999) Environmental enrichment inhibits spontaneous neuronal apoptosis and prevents neuronal injury in the rat. Nature Medicine, 5, 448-453.

9) During, M.J. and Janson, C.G. (1999) PNAs breach the blood-brain barrier? Nature Biotechnology 17, 753-754.

10) During, M.J., Symes, C.A., Lawlor, P.A., Lin, J., Dunning J., Fitzsimons, H.L., Poulsen, D., Leone, P., Xu, R., Dicker, B.L., Lipski, J. and Young, D., (2000) An Oral Vaccine Against NMDAR1 with efficacy in Experimental Stroke and Epilepsy. Science 287,1453-1460.

11) Leone, P., Janson, C.G., Bilianuk, L., Wang, J., Sorgi, F. McPhee, S., Haung, L., Matalon, R., Kaul. R., Freese, A., Young D., Xu, R., McPhee, S., Mee, E. and During, M.J. (2000) Apartoacylase gene transfer to the mammalian nervous system with therapeutic implications for Canavan Disease. Ann. Neurol. 48, 27-38.

12) Luo, J., Kaplitt, M.G., Fitzsimons, H.L., Zuzga, D., Liu, Y., Oshinsky, M.L. and During, M.J. (2002) Subthalamic GAD gene therapy in a Parkinson's Disease rat model. Science 298, 425-429.

13) During, M.J., Cao, L., Zuzga, D.S., Francis, J.S., Fitzsimons, H.L., Jiao, X., Bland, R.J., Klugmann, M., Banks, W.A., Drucker, D.J., Haile, C.N., (2003) Glucagon-like peptide-1 receptor involvement in learning and neuroprotection. Nature Medicine 9, 1173-1179.

14) Cannon, C.M., Abdallah, L., Tecott, L.H., During, M.J., Palmiter, R.D. (2004) Dysregulation of striatal dopamine signaling by amphetamine inhibits feeding in hungry mice. Neuron 44, 509-520.

15) Cao L, Jiao X, Zuzga DS, Liu Y, Fong DM, Young D, During MJ. (2004) VEGF links hippocampal activity with neurogenesis, learning and memory. Nat Genet. 36, 827-835.

16) Rashidian J, Iyirhiaro G, Aleyasin H, Rios M, Vincent I, Callaghan S, Bland RJ, Slack RS, During MJ, Park DS. (2005) Multiple cyclin-dependent kinases signals are critical mediators of ischemia/hypoxic neuronal death in vitro and in vivo. Proc Natl Acad Sci U S A. 102, 14080-5.

17) Klugmann, M., Leichtlein, C., Symes, W.C., Klaussner, B.C., Brooks, A.I., Young, D. During, M.J. (2005) A novel role of circadian transcription factor DBP in hippocampal plasticity. Mol. Cell. Neurosci. 31, 303-314.

18) Olson, V., Heusner, C.I., Bland, R.I., During, M.J., Weinshenker, D., Palmiter, R.D. (2006) Role of noradrenergic signaling by the nucleus tractus solitarious in mediating opiate reward. Science 311, 1017-1020.

19) Tappe, A., Klugmann, M, Luo, C., Hirlinger, D., Agarwal, N., Benrath, J., Ehrengruber, M., During, MJ, Kuner, R. (2006) Synaptic scaffolding protein Homer 1a protects against chronic inflammatory pain. Nat. Med. 12, 677-681.

20) Mouravlev, A., Dunning, J., Young, D., During, M.J. (2006) Somatic gene transfer of cyclic AMP response element-binding protein attenuates memory impairment in aging rats. Proc. Natl. Acad. Sci., USA 103, 4705-4710.

21) Fitzsimons, H.L., Riban V., Bland, R.J., Wendelken, J.L., Sapan, C.V., During, M.J. (2010) Biodistribution and safety assessment of rAAV-GAD following intrasubthalamic injection in the rat. Journal of Gene Medicine, 12: 385-389.

1) Kaplitt MG and Loewy AD, Editors. (1995) Viral Vectors: Gene Therapy and Neuroscience Applications. San Diego, CA: Academic Press.

2) Mobbs CV, Kaplitt MG and Pfaff DW (1997) "HIP-70/GRP58/Erp61/PLC-a/CPT: A single gene product and member of the protein disulfide isomerase gene family with thiol oxidoreductase activity subject to neuroendocrine regulation", in Prolyl Hydroxylase, Protein Disulfide Isomerase and Other Structurally Related Proteins (Guzman N, ed.) New York: Marcel Dekker, Inc.

3) Kaplitt MG and Loewy AD (1998) "Viral Vectors", in Fundamental Neuroscience (Bloom, et. al. eds) San Diego, CA: Academic Press.

4) Kaplitt MG and Lozano, AM (2001) Surgical drug delivery for neurodegenerative diseases. Clin Neurosurg 48:127-144.

5) Kaplitt MG, Hutchinson W and Lozano, AM (2001) "Target Localization in Movement Disorder Surgery", in Contemporary Clinical Neurology: Surgical Treatment of Parkinson's Disease and Other Movement Disorders (Tarsy, D. et. al., eds.) Totowa, NJ: Humana Press.

6) Kaplitt MG, Dostrovsky J, Hutchinson W and Lozano AM "Microelectrode Recording in Functional Neurosurgery" in Neurosurgery (Wilkins, RH and Rengachary, SS, eds.). New York: McGraw-Hill. In press.

7) Kaplitt MG and Loewy AD (2002) "Viral Vectors", in Fundamental Neuroscience, 2nd ed. (Bloom, et. al. eds) San Diego, CA: Academic Press.

8) Kaplitt MG, Rezai AR, Lozano A and Tasker R (2004) "Deep Brain Stimulation for Chronic Pain" in Youmans Neurological Surgery, 5th Edition (H.R. Winn, ed.). Philadelphia: Elsevier.

9) Kaplitt MG and During MJ, Editors (2006). Gene Transfer in the Central Nervous System: From Bench to Bedside. Amsterdam: Elsevier.

1) Mobbs CV, Kaplitt MG, Kow L-M, Pfaff DW (1991) PLC-a: A common mediator of the actionof estrogen and other hormones? Mol. Cell. Endocrinol. 80:C187-C191.

2) Kaplitt MG, Pfaus JG, Kleopoulos SP, Hanlon BA, Rabkin SD, Pfaff DW (1991) Expression of a functional foreign gene in adult mammalian brain following in vivo transfer via a herpes simplex virus type 1 defective viral vector. Mol. Cell. Neurosci. 2:320-330.

3) Kaplitt MG, Rabkin SD, Pfaff DW (1992) Molecular alterations in nerve cells: Direct manipulation and physiologic mediation. Curr. Top. Neuroendocrinol. 11:169-191.

4) Kaplitt MG, Kleopoulos SP, Pfaff DW, Mobbs CV. (1993) Estrogen induces HIP-70/PLC-amessenger RNA in the uterus and ventromedial hypothalamus. Endocrinology 133:99-104.

5) Brooks PJ, Kaplitt MG, Kleopoulos SP, Funabashi T, Mobbs CV, Pfaff DW. (1993) Sensitive detection of low abundance RNAs by in situ hybridization using single-stranded DNA probes produced by amplified primer extension labeling. J. Histochem. Cytochem. 41:1761-1766.

6) Kaplitt MG, Kwong AD, Kleopoulos SP, Mobbs CV, Rabkin SD, Pfaff DW. (1994) Preproenkephalin promoter yields region-specific and long-term expression in adult rat brain following in vivo transfer via a defective herpes simplex viral vector. Proc. Natl. Acad. Sci. USA, 91:8979-8983.

7) Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, OMalley K, During MJ (1994) Long-term expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Gen. 8:148-154.

8) Kaplitt MG, Tjuvajev J, Leib DA, Berk J, Rabkin SD, Posner JB, Pfaff DW, Blasberg RG. (1994) Mutant herpes simplex virus induced regression of tumors growing in immunocompetent rats. J. Neuro-oncology 19:137-142.

9) Verhaagen J, Hermens W, Oestricher B, Rabkin SD, Pfaff DW, Gispen WH, Kaplitt MG (1994) Expression of B-50 (GAP43) via a defective herpes simplex virus vector yields morphological changes in cultured non-neuronal cells. Mol. Brain Res. 26:26-36.

10) Dutt A, Kaplitt MG, Kow LM, Pfaff DW. (1994) Prolactin, CNS and Behavior, a critical review. Neuroendocrinol. 59:413-419.

11) Yin J, Kaplitt MG, Pfaff DW (1994) In situ PCR and in vivo detection of foreign gene expression in rat brain. Cell Vision 1:58-59.

12) Eizenberg O, Kaplitt MG, Eitan S, Pfaff DW, Hirschberg DL, Schwartz M. (1994) Linear dimeric interleukin-2 obtained by the use of a defective viral vector: conformation-activity relationship. Mol. Brain Res. 26:156-162.

13) Wood MJA, Byrnes A, Kaplitt MG, Pfaff DW, Rabkin SD, Charlton HD (1994) Specific pattern of defective HSV1 gene transfer in the adult central nervous system: Implications for gene targeting. Exp. Neurol. 130:127-140.

14) Rosenfeld MR, Meneses PM, Dalmau J, Drobnjak M, Cordon-Cardo C, Kaplitt MG (1995) Gene transfer of wild-type p53 results in restoration of tumor-suppressor function in a medulloblastoma cell line. Neurology 45:1533-1539.

15) Tjuvajev J, Gansbacher B, Desai R, Beattie B, Kaplitt MG, Matei C, Koutcher J, Gilboa E, Blasberg R. (1995) RG-2 glioma growth attenuation and severe brain edema caused by local production of interleukin-2 and interferon-gamma. Cancer Res. 55:1902-1910.

16) Holtmaat AJ, Hermens WT, Oestreicher AB, Gispen WH, Kaplitt MG, Verhaagen J. (1996) Efficient adenoviral vector directed expression of a foreign gene to neurons and sustentacular cells in the mouse olfactory neuroepithelium. Mol. Brain Res. 41:148-156.

17) Kaplitt M, Gouras GK, Makimura H, Jovanovic J, Sweeney D, Greengard P, Relkin NR, Gandy S. (1996) Apolipoprotein E, A beta-amyloid, and the molecular pathology of Alzheimer's disease. Therapeutic implications. Ann. N.Y. Acad. Sci. 802: 42-9.

18) Kaplitt MG, Xiao X, Samulski RJ, Li R, Ojamaa K, Klein IL, Makimura H, Kaplitt MJ, Strumpf RK, Diethrich EB (1996) Long-term gene transfer in porcine myocardium after coronary infusion of an adeno-associated virus vector. Ann. Thoracic Surg. 62(6): 1669-76.

19) Vogt L, Giger RJ, Ziegler U, Kunz B, Buchstaller A, Hermens W, Kaplitt MG, Rosenfeld MR, Pfaff DW, Verhagen J, Sonderegger P (1996) Continuous renewal of the axonal pathway sensor apparatus by insertion of new sensor molecules into the growth cone membrane. Current Biology 6(9): 1153-8.

20) Freese A, Stern M, Kaplitt MG, O'Connor WM, Abbey MV, O'Connor MJ, During MJ (1996) Prospects for gene therapy in Parkinson's disease. Movement Disorders 11:469-488.

21) Freese A, During MJ, Davidson BJ, Gennarelli TA, Kaplitt MG, Flamm ES, Snyder PJ (1996) Transfection of human lactotroph adenoma cells with an adenovirus vector expressing tyrosine hydroxylase decreases prolactin release. J. Clin. Endocrinol. Metab. 81: 2401-4.

22) Freese A, Kaplitt MG, WM OC, Abbey M, Langer D, Leone P, MJ OC, During MJ (1997) Direct gene transfer into human epileptogenic hippocampal tissue with an adeno-associated virus vector: implications for a gene therapy approach to epilepsy. Epilepsia 38:759-66.

23) Holtmaat AW, Hermens V, Sonnemans MAF, Giger RJ, Van Leeuwen FW, Kaplitt MG, Oestreicher AB, Gispen WH, Verhaagen J (1997) Adenoviral Vector-Mediated Expression of B-50/GAP-43 Induces Alterations in the Membrane Organization of Olfactory Axon Terminals In Vivo. J Neurosci 17:6575-86.

24) Kaplitt MG, Makimura H (1997) Defective viral vectors as agents for gene transfer in the nervous system. J Neurosci Methods 71:125-32.

25) O'Connor WM, Davidson BL, Kaplitt MG, Abbey MV, During MJ, Leone P, Langer D, MJ O'Conner and Freese A (1997) Adenovirus vector-mediated gene transfer into human epileptogenic brain slices: prospects for gene therapy in epilepsy. Exp Neurol 148:167-78.

26) Rosenfeld MR, Bergman I, Schramm L, Griffin JA, Kaplitt MG, Meneses PI (1997). Adeno-associated viral vector gene transfer into leptomeningeal xenografts. J Neurooncol 34(2): 139-44.

27) Sugiyama A, Hattori S, Tanaka S, Isoda F, Kleopoulos S, Rosenfeld M, Kaplitt M, Sekihara H, Mobbs C (1997) Defective adeno-associated viral-mediated transfection of insulin gene by direct injection into liver parenchyma decreases blood glucose of diabetic mice. Horm Metab Res 29:599-603.

28) During MJ, Samulski RJ, Elsworth JD, Kaplitt MG, Leone P, Xiao X, Li J, Freese A, Taylor JR, Roth RH, Sladek JR, O'Malley KL, Redmond DE (1998) In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Therapy 5:820-827.

29) Kaplitt MG, Darakchiev BJ, During MJ (1998) Prospects for gene therapy in pediatric neurosurgery. Pediatr Neurosurg 28:3-14.

30) During MJ, Xu R, Young D, Kaplitt MG, Sherwin RS, Leone P (1998) Peroral gene therapy of lactose intolerance using an adeno-associated virus vector. Nat Med 4:1131-1135.

31) Yin J, Kaplitt MG, Kwong AD, Pfaff DW (1998) In situ PCR for in vivo detection of foreign genes transferred into rat brain. Brain Res 783:347-354.

32) Kanno H, Hattori S, Sato H, Murata H, Huang FH, Hayashi A, Suzuki N, Yamamoto I, Kawamoto S, Minami M, Miyatake S, Shuin T, Kaplitt MG (1999) Experimental gene therapy against subcutaneously implanted glioma with a herpes simplex virus-defective vector expressing interferon-gamma. Cancer Gene Ther 6:147-154.

33) Thomas DL, Shin S, Jiang BH, Vogel H, Ross MA, Kaplitt M, Shenk TE, Javier RT (1999) Early region 1 transforming functions are dispensable for mammary tumorigenesis by human adenovirus type 9. J Virol 73:3071-3079.

34) Bilsky MH, Downey RJ, Kaplitt MG, Elowitz EH, Rusch VW (2001) Tension pneumocephalus resulting from iatrogenic subarachnoid-pleural fistulae: report of three cases. Ann Thorac Surg 71:455-457.

35) During MJ, Kaplitt MG, Stern MB, Eidelberg D (2001) Subthalamic GAD gene transfer in Parkinson's disease patients who are candidates for deep brain stimulation. Hum Gene Ther 12:1589-1591.

36) Luo J, Kaplitt MG, Fitzsimons HL, Zuzga D, Raniga S, Oshinsky M, During MJ (2002) Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science 298:425-429.

37) Moussatov S, Roberts J, Pfaff D and Kaplitt MG (2002) A cis-acting element that directs exclusively circular adeno-associated virus replication and packaging. J. Virol. 76:12792-12802.

38) Betchen S and Kaplitt MG (2003) Current and future surgical therapies for Parkinson's Disease. Curr. Op. Neurol. 16:487-494.

39) Scott RE, Wu-Peng XS, Kaplitt MG, Pfaff DW (2003) Gene transfer and in vivo promoter analysis of the rat progesterone receptor using a herpes simplex virus viral vector. Mol. Brain Res. 114:91-100.

40) Hamani C, Saint-Cyr JA, Fraser J, Kaplitt M, Lozano AM (2004) The subthalamic nucleus in the context of movement disorders. Brain 127:4-20.

41) Musatov S, Roberts J, Brooks AI, Pena J, Betchen S, Pfaff DW and Kaplitt MG (2004). The PTEN anti-oncogene negatively regulates the neuronal phenotype in PC12 cells. Proc. Natl. Acad. Sci. USA, 101:3627-3634.

42) Crystal RG, Sondhi D, Hackett NR, Kaminsky SM, Worgall S, Stieg P, Souweidane M, Hosain S, Heier L, Ballon D, Dinner M, Wisniewski K, Kaplitt M, Greenwald BM, Howell JD, Strybing K, Dyke J, Voss H (2004) Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis. Hum. Gen. Ther. 15:1131-1154.

43) Arkin LM, Sondhi D, Worgall S, Suh LH, Hackett NR, Kaminsky SM, Hosain SA, Souweidane MM, Kaplitt MG, Dyke JP, Heier LA, Ballon DJ, Shungu DC, Wisniewski KE, Greenwald BM, Hollmann C, Crystal RG (2005) Confronting the issues of therapeutic misconception, enrollment decisions and personal motives in genetic medicine-based clinical research studies for fatal disorders. Hum. Gene Ther. 16:1028-1036.

44) Betchen S, Mousatov S, Roberts J, Pena J, Kaplitt MG (2006) PTEN inhibits adrenomedullin expression and function in glioma cells. J Neuroonc. 79:117-123.

45) Cooper L, Chan D, Roediger F, Shaffer B, Fraser J, Musatov S, Selesnick S, Kaplitt MG. (2006) AAV-Mediated Delivery of the Caspase Inhibitor XIAP Protects Against Cisplatin Ototoxicity. Otology and Neurot., 27:484-490.

46) Musatov S, Chen W, Pfaff DW, Kaplitt MG, Ogawa S (2006) RNAi-mediated silencing of estrogen receptor a in the ventromedial nucleus of the hypothalamus abolishes female sexual behaviors. Proc. Natl. Acad. Sci. USA, 103:10456-1460.

47) Asanuma K, Tang C, Ma Y, Dhawan V, Mattis P, Edwards C, Kaplitt MG, Feigin A, Eidelberg D (2006) Network modulation in the treatment of Parkinson's disease. Brain, In press.

48) Chan D, Lieberman D, Mousatov S, Selesnick S, Kaplitt MG (2006) Protection against cisplatin-induced ototoxicity by AAV-mediated delivery of the X-linked inhibitor of apoptosis (XIAP) is not dependent on caspase inhibition. Otology and Neurot., In press.

49) Emborg ME, Carbon M, Holden JE, During MJ, Ma Y, Tang C, Moirano J, Fitzsimons HL, Roitberg BZ, Tuccar E, Roberts A, Kaplitt MG, Eidelberg D. (2007) Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism. Jour. of Cereb Blood Flow & Metab. 27:501-509.