Molecular pharmaceutics represents interdisciplinary specialties within the pharmaceutical sciences and encompasses a range of scientific endeavors, including:

• The design, fabrication, and evaluation of dosage forms and delivery strategies for conventional and biotechnology-based therapeutic agents
• Elucidation of barriers to systemic and target-specific drug delivery
• Determination of the ability of pharmacologic agents to reach the relevant site of biologic effect
• Determination of the time course of biologic activity

These areas of specialization represent critical steps in the development of new therapeutic agents, the evaluation of new and existing agents in a variety of settings (e.g., potential interactions with other agents or alterations secondary to physiologic changes associated with disease processes), and the optimal clinical use of pharmacologic agents.

See the division faculty page for more information on our faculty and their research

Recent Grants Received

• Novel Nanoparticles for siRNA Delivery
  Leaf Huang, National Cancer Institute, 4/1/2010 - 1/31/2015  $1,250,000
• Re-Activation of Maspin Tumore Suppressor Gene By Designed Transciption Factors
  Leaf Huang (P.I. Blancafort), NIH, 09/30/2009 - 9/29/2011  $182,733
• BCG Supporting Research
  Anthony J. Hickey, Harvard University, 6/01/2009 - 6/30/ 2010  $342,918
• Interaction of Cationic Lipids with Dendritic Cells
  Leaf Huang, National Cancer Institute, 3/05/08 - 2/28/13, $1,506,414
• LPD Nanparticles in Anti-Cancer Therapy
  Leaf Huang, National Cancer Institute, 3/07/08 - 2/28/13, $1,505,096
• Computational Models and High-Throughput Cellular-Based Toxicity Assays for Predictive Nanotoxicology
  Russell Mumper (P.I. Tropsha) Univ. of Arizona, 4/1/2009- 12/31/2010  $61,656
• Studies to Elucidate the Properties of Antisense Oligonucleotides in Aqueous Solution
  Russell Mumper, Isis Pharmaceuticals, Inc., 9/22/2009 - 8/31/2010,  $48,918
• Myostatin Inhibition in DMD Dogs by Gene Transfer
  Xiao Xiao, NIAMS, 7/01/2009 - 6/30-2014, $1,125,000
• Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center ARRA
  Xiao Xiao (P.I. Samulski), NIAMS, 9/05/2008 - 8/31/2010, $956,000

Recent Publications

• M.J. Cho
  Palma, E. and Cho, M.J.: Improved Systemic Pharmacokinetics, Biodistribution, and Antitumor Activity of CpG Oligodeoxynucleotides Complexed to Endogenous Antibodies in Vivo. J. Controlled Release 120:95-103, 2007.
  
  Gratton, S., Pohlhaus, P.D., Lee, J., Cho, M.J., and DeSimone, J.M.: Nanofabricated Particles for Engineered Drug Therapies: A Preliminary Biodistribution Study of PRINT Nanoparticles, J. Controlled Release 121:10-18, 2007.
• Anthony Hickey
  Shi, S., Ashley, E.S., Alexander, B.D., Hickey, A.J.  Initial characterization of micafungin pulmonary delivery via two different nebulizers and multivariate data analysis of aerosol mass distribution profiles.  AAPS PharmSciTech 2009, 10 (1): 129-37.
  
  Wang, C. Muttil, P., Lu, D., Beltran-Terres, A., Garcia-Contreras, L., Hickey, A.J.  Screening for potential adjuvants adminstered by the pulmonary route for tuberculosis vaccines.  AAPS J 2009 March, 11 (1): 139-47.
  
  Sung, J.C. Padilla, D.J., Garcia-Contreras, L., VerBerkmoes, J.L., Durbin, D., Peloquin, C.A., Elbert, K.J., Hickey, A.J., and Edwards, D.A.  Formulation and pharmacokinetics of self-assembled rifampicin nanoparticle systems for pulmonary delivery. Pharm. Res. 2009, 26(8): 1847-55.
  
  Hickey, A.J., and Garmise, R.J.  Dry powder nasal vaccines as an alternative to needle-based delivery.  Crit. Rev. Ther Drug Carrier Syste. 2009, 26 (1): 1-27.
  
  Shi, S., HIckey, A.J. Multivariate data analysis as a semi-quantitative tool for interpretive evaluation of comparability or equivalence of aerodynamic particle size distribution profiles. AAPS PharmSciTech 2009, 10(4): 1113-20. 
  
  Shi, S., Yu, L., Sun, D., Liu, J. Hickey, A.J.  Rational design of multiple TB antigens TB10.$ and TB10.4-Ag85B as subunit vaccine candidates against Mycobacterium tuberculosis.  Pharm Res 2010, 27(2): 224-34.
• Leaf Huang
  Chen, Y., Sen J., Bathula, S., Yang, Q., Fittipaldi, R., and Huang, L. Novel Cationic Lipid That Delivers siRNA and Enhances Therapeutic Effect in Lung Cancer Cells.  Molecular Pharmaceutics. 6:696-705, 2009. (PMCID: PMC2763490)
  
  Li, S-D, Huang, L. Nanoparticles Evading the Reticuloendothelial System: Role of the Supported Bilayer. Biochim. Biophys. Acta – Biomembranes. 1788:2259 – 2266, 2009  (PMCID: PMC2757503)
  
  Li, J., Chen, Y-C., Tseng, Y-C., Mozumdar, S., and Huang, L. Biodegradable Calcium Phosphate Nanoparticle with Lipid Coating for Systemic siRNA Delivery. Journal of Controlled Release. 142:416-421, 2010.
  
  Chen, Y., Wu, J.J., and Huang, L.  Nanoparticles Targeted with NGR Motif Deliver c-Myc siRNA and Doxorubicin for Anticancer Therapy.  Molecular Therapy 18(4): 828 – 834, 2010.
  
  Liu, F., Frick, A., Yuan, X., and Huang, L. Dysopsonin Activity of Serum DNA-Binding Proteins Favorable for Gene Delivery. J. Pharm. Expt. Ther. Published on October 28, 2009 as DOI:10.1124/jpet.109.159541
  
  Deng, J., Huang, L., and Liu, F. Understanding the Structure and Stability of Paclitaxel Nanocrystals.  International Journal of Pharmaceutics. 2010 Feb 16 (Epub ahead of print).
  
  307.    Liu, F., Zhang, Y., Conwell, C., Zhang, Y., Bathula, S., and Huang, L. Targeted Cancer Therapy with Novel High Drug-loading Nanocrystals. Journal of Pharmaceutical Sciences. (In press).
  
  Deng, J., Huang, L., and Liu, F.  Extraction Issues of Paclitaxel in Nanocrystals.  Journal of Biomedical Nanotechnology. (In press).
  
  Liu, Y., and Huang, L. Designed Lipids Advance Systemic siRNA Delivery.  Molecular Therapy 18(4): 669 – 670, 2010.
  
  Liu, Y., Huang, L., and Liu, F. Paclitaxel Nanocrystals for Overcoming Multidrug Resistance in Cancer. Molecular Pharmaceutics. (In press).
  
  Chen, Y., Bathula, R., Li, J., and Huang, L. Multifunctional Nanoparticles Delivering siRNA and Doxorubicin Overcome Drug Resistance in Cancer. Journal of Biological Chemistry. (In press).
  
  Nakagawa, O., Ming, X., Huang, L.,  and Juliano, R.L. Targeted Intracellular Delivery of Antisense Oligonucleotides Via Conjugation With Small Molecule Ligands. JACS. (In press)
• Michael Jay
  MD Howard, M Jay, TD Dziubla and X. Lu.  PEGylation of Nanocarrier Drug Delivery Systems: State of the Art.  J Biomed Nanotechnology 4(2):133-148 (2008).
  
  Lu X, Howard MD, Talbert DR, Rinehart JJ, Jay M and Leggas M. Nanoparticles containing anti-inflammatory agents as chemotherapy adjuvants II: role of plasma esterases in drug release. AAPS J. 11:120-2 (2009).
  
  Sutthanut K, Lu X, Jay M and Sripanidkulchai B.  Solid lipid nanoparticles for topical administration of kaempferia parviflora extracts. J Biomed Nanotechnology. 5:224–232 (2009).
• Rudy Juliano
  Alam R, Ming X, Dixit V, Fisher M, Chen X and Juliano RL The biological effect of an antisense oligonucleotide depends on its route of endocytosis and trafficking. Oligonucleotides, in press, 2009.
  
  Juliano RL, Bauman J, Kang H, Ming X. Biological barriers to therapy with antisense and siRNA oligonucleotides. Molecular Pharmaceutics, 6: 686-95, 2009 (review).
  
  Fisher Ms, Abramove M, Van Aerschot A, Rozenski J, Dixit V, Herdewijn P and Juliano R. Biological effects of hexitol and altritol-modified siRNAs targeting B-Raf. Eur J Pharmacol, 606:38-44, 2009
• Richard J. Kowalsky
  Bandarenko N, Cancelas J, Synder EL, Hay SN, Rugg N, Corda T, Joines AD, Gormas JF, Kowalsky RJ, Rose M, Rose L, Foley J, Popovsky MA. Successful In Vivo Recovery and Extended Storage of Additive Solution (AS) – 5 Red Blood Cells after Deglycerolization and Resuspension in AS-3 for 15 days with an Automated Closed System. Transfusion 47: 680-686, 2007.
  
  Schultz MK, Cessna JT, Norenberg JP, Anderson TL, Ponto JA, Petry N, Kowalsky RJ, Palmer                             M, Beinlich U, Divgi C. A Performance Evaluation of 90Y Dose Calibrator Measurements in                                Nuclear Pharmacies and Clinics in the United States. Appl Rad Isot 2008; 66: 252-260.
• Russell Mumper
  AS Holpuch , GJ Hummel, M Tong, GA Seghi, P Pei, P Ma, RJ Mumper, and SR Mallery. Nanoparticles for Local Drug Delivery to the Oral Mucosa: Proof of Principle Studies.  Pharmaceutical Research. (2010 Mar 31). [E-pub ahead of print].
  
  S Wadhwa, and RJ Mumper. Intracellular Delivery of the Reactive Oxygen Species Generating Agent d-Penicillamine upon Conjugation to Poly-I-glutamic Acid. Molecular Pharmaceutics.  (2010 Apr 6). [E-pub ahead of print].
  
  W Yan, A Jain, R  O’Carra, JG  Woodward, W Li, G Li, A Nath and RJ Mumper.  Lipid Nanoparticles with Accessible Nickel as a Vaccine Delivery System for Single and Multiple His-tagged HIV Antigens.  HIV/AIDS - Research and Palliative Care.  (2009) 1:1-11.
  
  X Dong, CA Mattingly, M Tseng, M Cho, Y Liu, VR Adams, and RJ Mumper. Doxorubicin and Paclitaxel-loaded Lipid-based Nanoparticles Overcome Multi-Drug Resistance By Inhibiting P-gp via ATP Depletion.  Cancer Research. (2009) 69:3918-3926.
  
  P Ma, X Dong, CL Swadley, A Gupte, M Leggas, HC Ledebur, and RJ Mumper. Development of Idarubicin and Doxorubicin Solid Lipid Nanoparticles to Overcome Pgp–mediated Multiple Drug Resistance in Leukemia.  J. Biomed. Nanotech.  (2009) 5:151-161.
  
  CM Ugalde, Z Liu, C Ren, KK Chan, KA Rodrigo, Y Ling, PE Larsen, GE Chacon, GD Stoner, RJ Mumper, HW Fields, SR RMallery.  Distribution of Anthocyanins Delivered from a Bioadhesive Black Raspberry Gel Following Topical Intraoral Application in Normal Healthy Volunteers.  Pharm. Res. (2009) 26:977-986.
  
  RJ Mumper, MA Bell, DR Worthen, RA Cone, GR Lewis, and TR Moench.  Formulating a Sulfonated Anti-Viral Dendrimer in a Vaginal Microbicidal Gel having Dual Mechanisms of Action.  Drug Dev. Ind. Pharm.  (2009) 35:515-524.
  
  X Dong, CA Mattingly, M Tseng, M Cho, VR Adams, and RJ Mumper.  Development of New Lipid-Based Paclitaxel Nanoparticles Using Sequential Simplex Optimization.  Eur. J. Pharm. Biopharm.  (2009) 72:9-17.
  
  J Dai, A Gupte, L Gates, and RJ Mumper. A Comprehensive Study of Anthocyanin-Rich Extracts from Selected Blackberry Cultivars: Extraction Methods, Stability, Anticancer Properties and Mechanisms.  Food and Chemical Tox. (2009) 47:837-847.
  
  

• Philip C. Smith
  RL  Hawke, SJ Schrieber, TA Soule, Z Wen, PC Smith, KR Reddy, AS Wahed, SH Belle, NH Afdhal, VJ Navarro, J Berman, QY Liu, E Doo, MW  Fried.   Silymarin ascending multiple oral dosing phase I study in noncirrhotic patients with chronic hepatitis C.  J Clin Pharmacol. 50(4):434-439 (2010).
  
  JQ Dong, PC Smith.  Glucuronidation and covalent protein binding of benoxaprofen and flunoxaprofen in sandwich-cultured rat and human hepatocytes.  Drug Metab Dispos. 37(12):2314-2322 (2009).
  
  MS Joy, T Hilliard, Y Hu, SL Hogan, J Wang, RJ Falk RJ, PC Smith.  Influence of clinical and demographic variables on mycophenolic acid pharmacokinetics in antineutrophil cytoplasmic antibody-associated vasculitis.  Ann Pharmacother. 43(6):1020-1027 (2009).
  
  AH Harrill, PB Watkins, S Su, PK Ross, DE Harbourt, IM Stylianou, GA Boorman, MW Russo, RS Sackler, SC Harris, PC Smith, R Tennant, M Bogue, K Paigen, C Harris, T Contractor. T Wiltshire, I Rusyn, DW Threadgill.  Mouse population-guided resequencing reveals that variants in CD44 contribute to acetaminophen-induced liver injury in humans.   Genome Res. 19(9):1507-1515 (2009).
  
  MS Joy, T Hilliard, Y Hu, SL Hogan, MA Dooley, RJ Falk, PC Smith.  Pharmacokinetics of mycophenolic acid in patients with lupus nephritis.   Pharmacotherapy. Jan;29(1): 7-16 (2009).
  
  

• Xiao Xiao
  Chen CC, Sun CP, Ma HI, Fang CC, Wu PY, Xiao X, Tao MH.(2009) Comparative study of anti-hepatitis B virus RNA interference by double-stranded adeno-associated virus serotypes 7, 8, and 9. Molecular Therapy. 2009 Feb;17:352-9.
  
  L. Yang, J. Jiang, LM. Drouin, M. Agbandje-Mckenna, C. Chen, C. Qiao, X. Hu, D. Wang, J. Li, Xiao X.(2009) A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection, Proc. Natl. Acad. Sci. USA, 106 (10):3946-3951.
  
  A. Mejat, V. Decostre, J, Li, L. Renou, A. Kesari, D. Hantaï, C L. Stewart, X. Xiao, E. Hoffman, G. Bonne & T. Misteli (2009) Lamin A/C mediates neuromuscular junction defects in Emery-Dreifuss Muscular Dystrophy, Journal of Cell biology. 184:31-44.
  
  C. Yu, Z. Yuan, Z. Cao, B. Wang, J. Li & Xiao X. (2009) A muscle-targeting peptide displayed on AAV2 improves muscle tropism upon systemic delivery. Gene Therapy. 16 :953-962.
  
  Koppanati, BM; Li, J; Xiao, X; Clemens, PR (2009) Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders. Gene Therapy 16:1130-1137.  
  
  Tsai ML, Horng CT, Chen SL, Xiao X, Wang CH, Tsao YP.(2009) Suppression of experimental uveitis by a recombinant adeno-associated virus vector encoding interleukin-1 receptor antagonist. Mol Vis. 2009 Aug 8;15:1542-52.
  
  Pan J, Zhang Q, Zhou J, Ma D, Xiao X, Wang DW.(2009) Recombinant adeno-associatedvirus encoding Epstein-Barr virus latent membrane proteins fused with heat shock protein as a potential vaccine for nasopharyngeal carcinoma. Mol Cancer Therapy 8(9):2754-61.
  
  Zheng H, Qiao C, Wang CH, Li J, Li J, Yuan Z, Zhang C, Xiao X. .(2009)  Efficient Retrograde Transport of AAV8 to Spinal Cord and Dorsal Root Ganglion after Vector Delivery in Muscle. Hum Gene Therapy. EPub, Aug 28.
  
  Zhou L, Zhu T, Ye X, Yang L, Wang B, Liang X, Lu L, Tsao YP, Chen SL, Li J, Xiao X. (2009) Long-term protection against HPV E7-positive tumor by a single vaccination of AAV vectors encoding a fusion protein of inactivated E7 of HPV 16/18 and heat shock protein 70. Hum Gene Therapy. EPub, Aug 28.
  
  Yan W, Wang P, Zhao CX, Tang J, Xiao X, Wang DW. .(2009)  Decorin Gene Delivery Inhibits Cardiac Fibrosis in Spontaneously Hypertensive Rats by Modulation of Transforming Growth Factor-beta/Smad and p38 Mitogen-Activated Protein Kinase Signaling Pathways. Hum Gene Therapy. EPub, Aug 21.
  
  Pichavant C.,  P. Chapdelaine, DG. Cerri, JC. Dominique, SP. Quenneville, D. Skuk1, JN. Kornegay, J. Bizario, X. Xiao, Jacques P Tremblay (2010) Expression of Dog Microdystrophin in Mouse and Dog Muscles by Gene Therapy, Molecular Therapy, in press.
  
  JR. Mendell, K. Campbell, L. Rodino-Klapac, Zarife Sahenk, C. Shilling, S. Lewis, D. Bowles, S. Gray, C. Li, G. Galloway, V. Malik1, B. Coley, R. Clark, J. Li, X. Xiao, J. Samulski, SW. McPhee, RJ Samulski, CM. Walker (2010) Transient Expression of a Therapeutic Dystrophin Transgene in Duchenne Muscular Dystrophy Revealed by T Cell Mediated Immunity, New England J. Medicine., In press.
  
  CH. Lo, CM. Chang, SW. Tang, WY. Pan, CC. Fang, Y. Chen, PY. Wu, KY. Chen, HI. Ma, X. Xiao, MH. Tao (2010) Differential Antitumor Effect of Interleukin 12 Family Cytokines on Orthotopic Hepatocellular Carcinoma  J. Gene Medicine in press.
  
  JF Chen, Y Tao, J Li, Z Yan, X Xiao, DZ Wang (2010) MicroRNA-1 and miR-206 Regulate Skeletal Muscle Satellite Cell Proliferation and Differentiation by Repressing Pax7  J. Cell Biology, in press.
  
  JN. Kornegay, J Li, JR. Bogan, DJ. Bogan,  CL. Chen, H Zheng , CP Qiao, JF. Howard, and X Xiao (2010) Widespread Long-term Muscle Expression of an AAV-9 Human Mini-dystrophin vector after Intravenous Injection in neonatal Golden Retriever Muscular Dystrophy Dogs Molecular Therapy accepted for publication.
  
  Koppanati, BM., J. Li, B. Wang, J. Watchko, Xiao, X; Clemens, PR (2010) Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene Gene Therapy accepted for publication.
  
  CH. Wang, YM Chan, H Zheng, H Zhou, P Lu , E Keramaris-Vrantsis, CP Qiao, B Xiao, JG Jiang, J Li, HI Ma, Q Lu, X Xiao (2010) Post-natal knockdown of Fukutin related protein expression in muscle by long-term RNA interference induces dystrophic pathology, Human Molecular Genetics, under revision.
  
  HI Ma, HA Shui, CH Wang, CY Wang, DY Huang, SH Chiou, HM Chen, J Li, SY Cheng, X Xiao (2010) Intratumoral decorin gene delivery by AAV vector inhibits brain glioblastomas and prolongs survival of animals by inducing cell differentiation, Human Gene Therapy, submitted.