Next generation delivery system for proteins and genes of therapeutic purpose: Why and how?

BioMed Research International

Volumn 2014, Issue , 2014, Pages

  Sharma, Ashish Ranjan ^a   Kundu, Shyamal Kumar ^b   Nam, Ju Suk ^a   Sharma, Garima ^a   Doss, C George Priya ^c   Lee, Sang Soo ^a   Chakraborty, Chiranjib ^a,b  

^a Hallym University   (South Korea)

^b GALGOTIAS UNIVERSITY   (India)

^c VIT UNIVERSITY   (India)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; DRUG CARRIER; HETASTARCH; LIPOSOME; MACROGOL; NANOPARTICLE; PEPTIDE; PROTEIN; RNA; SMALL INTERFERING RNA; VIRUS VECTOR;

DRUG CONJUGATION; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG FORMULATION; DRUG SAFETY; GENE DELIVERY SYSTEM; GENE THERAPY; REVIEW; SIALYLATION; CHEMISTRY; GENE TRANSFER; GENETICS; HUMAN;

DRUG DELIVERY SYSTEMS; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HUMANS; LIPOSOMES; NANOPARTICLES; PROTEINS;

EID: 84924957605     PISSN: 23146133     EISSN: 23146141     Source Type: Journal    
DOI: 10.1155/2014/327950     Document Type: Review

References (133)

1. B. Leader, Q. J. Baca, and D. E. Golan, "Protein therapeutics: a summary and pharmacological classification," Nature Reviews Drug Discovery, vol. 7, no. 1, pp. 21-39, 2008.
2. C. Chakraborty and R. Jhingan, Protein Based Drugs: Techno Commercial Approach, Biotech Books, New Delhi, India, 2005.
3. F. G. Banting, C. H. Best, J. B. Collip, W. R. Campbell, and A. A. Fletcher, "Pancreatic extracts in the treatment of diabetes mellitus: preliminary report 1922," Canadian Medical Association Journal, vol. 145, no. 10, pp. 1281-1286, 1991.
4. J. Pickup, "Human insulin," British Medical Journal, vol. 292, no. 6514, pp. 155-157, 1986.
5. G. Schernthaner, "Immunogenicity and allergenic potential of animal and human insulins," Diabetes Care, vol. 16, no. 3, pp. 155-165, 1993.
6. D. V. Goeddel, D. G. Kleid, and F. Bolivar, "Expression in Escherichia coli of chemically synthesized genes for human insulin," Proceedings of the National Academy of Sciences of the United States of America, vol. 76, no. 1, pp. 106-110, 1979.
7. H. Keen, A. Glynne, and J. C. Pickup, "Human insulin produced by recombinant DNA technology: safety and hypoglycaemic potency in healthy men," The Lancet, vol. 2, no. 8191, pp. 398-401, 1980.
8. A. J. L. Clark, R.O. Adeniyi-Jones, G. Knight et al., "Biosynthetic human insulin in the treatment of diabetes," The Lancet, vol. 2, no. 8294, pp. 354-357, 1982.
9. C. Chakraborty, Production Technology of Recombinant Therapeutic Proteins, Biotech Books, New Delhi, India, 2004.
10. G. Walsh, "Biopharmaceutical benchmarks 2010," Nature Biotechnology, vol. 28, no. 9, pp. 917-924, 2010.
11. C. Chakraborty and G. Agoramoorthy, "A special report on India's biotech scenario: advancement in biopharmaceutical and health care sectors," Biotechnology Advances, vol. 28, no. 1, pp. 1-6, 2010.
12. T. Friedmann and R. Roblin, "Gene therapy for human genetic disease?" Science, vol. 175, no. 4025, pp. 949-955, 1972.
13. M. L. Edelstein, M. R. Abedi, and J. Wixon, "Gene therapy clinical trials worldwide to 2007 - an update," Journal of Gene Medicine, vol. 9, no. 10, pp. 833-842, 2007.
14. S. L. Ginn, I. E. Alexander, M. L. Edelstein, M. R. Abedi, and J. Wixon, "Gene therapy clinical trials worldwide to 2012 - an update," Journal of Gene Medicine, vol. 15, no. 2, pp. 65-77, 2013.
15. O. Kayser and H. Warzecha, Eds., Pharmaceutical Biotechnology: Drug Discovery and Clinical Applications, John Wiley & Sons, 2012.
16. T. M. Allen and P. R.Cullis, "Drug delivery systems: entering the mainstream," Science, vol. 303, no. 5665, pp. 1818-1822, 2004.
17. C. Chakraborty, S. Pal, G. P. Doss, Z. Wen, and C. Lin, "Nanoparticles as "smart" pharmaceutical delivery," Frontiers in Bioscience, vol. 18, no. 3, pp. 1030-1050, 2013.
18. K. Fletcher, "Drug delivery: strategies and technologies," Pharmaceutical Science & Technology Today, vol. 1, no. 2, pp. 49-51, 1998.
19. A. T. Florence, "New drug delivery systems," Chemistry and Industry, no. 24, pp. 1000-1004, 1993.
20. R. Langer, "Newmethods of drug delivery," Science, vol. 249, no. 4976, pp. 1527-1533, 1990.
21. R. K. Verma and S. Garg, "Current status of drug delivery technologies and future directions," Pharmaceutical Technology, vol. 25, no. 2, pp. 1-14, 2001.
22. L. R. Brown, "Commercial challenges of protein drug delivery," Expert Opinion on Drug Delivery, vol. 2, no. 1, pp. 29-42, 2005.
23. J. M. Reichert, "Trends in development and approval times for new therapeutics in the United States," Nature Reviews Drug Discovery, vol. 2, no. 9, pp. 695-702, 2003.
24. Y. Seow and M. J. Wood, "Biological gene delivery vehicles: beyond viral vectors," Molecular Therapy, vol. 17, no. 5, pp. 767-777, 2009.
25. D. S. Dimitrov, "Therapeutic proteins," Methods in Molecular Biology, vol. 899, pp. 1-26, 2012.
26. D. S. Pisal, M. P. Kosloski, and S. V. Balu-Iyer, "Delivery of therapeutic proteins," Journal of Pharmaceutical Sciences, vol. 99, no. 6, pp. 2557-2575, 2010.
27. M. A. Kay, "State-of-the-art gene-based therapies: the road ahead," Nature Reviews Genetics, vol. 12, no. 5, pp. 316-328, 2011.
28. D. B. Kohn, "Update on gene therapy for immunodeficiencies," Clinical Immunology, vol. 135, no. 2, pp. 247-254, 2010.
29. F. Depreter, G. Pilcer, and K. Amighi, "Inhaled proteins: challenges and perspectives," International Journal of Pharmaceutics, vol. 447, no. 1-2, pp. 251-280, 2013.
30. P. Zhang, K. F. Chan, R. Haryadi, M. Bardor, and Z. Song, "CHO glycosylation mutants as potential host cells to produce therapeutic proteins with enhanced efficacy," Advances in Biochemical Engineering/Biotechnology, vol. 131, pp. 63-87, 2013.
31. G. Gregoriadis, S. Jain, I. Papaioannou, and ETAL, "Improving the therapeutic efficacy of peptides and proteins: a role for polysialic acids," International Journal of Pharmaceutics, vol. 300, no. 1-2, pp. 125-130, 2005.
32. U. V. Banakar, "Advances and opportunities in delivery of therapeutic proteins and peptides," Journal of Biomaterials Applications, vol. 11, no. 4, pp. 377-429, 1997.
33. Y. Levy, M. S. Hershfield, C. Fernandez-Mejia et al., "Adenosine deaminase deficiency with late onset of recurrent infections: response to treatment with polyethylene glycol-modified adenosine deaminase," Journal of Pediatrics, vol. 113, no. 2, pp. 312-317, 1988.
34. S. Coura Rdos and N. B. Nardi, "The state of the art of adeno-associated virus based vectors in gene therapy," Virology Journal, vol. 7, article 12, 2007.
35. S.Hacein-Bey-Abina, A. Garrigue, G. P.Wang et al., "Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1," Journal of Clinical Investigation, vol. 118, no. 9, pp. 3132-3142, 2008.
36. B. Kedika and S. V. Patri, "Benzothiazole head group based cationic lipids: synthesis and application for gene delivery," European Journal of Medicinal Chemistry, vol. 74, pp. 703-716, 2013.
37. J. Sánchez-Nieves, P. Fransen, D. Pulido et al., "Amphiphilic cationic carbosilane-PEG dendrimers: synthesis and applications in gene therapy," European Journal of Medicinal Chemistry, vol. 76, pp. 43-52, 2014.
38. J. Swaminathan and C. Ehrhardt, "Liposomal delivery of proteins and peptides," Expert Opinion on Drug Delivery, vol. 9, no. 12, pp. 1489-1503, 2012.
39. H. M. Patel and B. E. Ryman, "Oral administration of insulin by encapsulation within liposomes," The FEBS Letters, vol. 62, no. 1, pp. 60-63, 1976.
40. H. M. Patel, R. W. Stevenson, J. A. Parsons, and B. E. Ryman, "Use of liposomes to aid intestinal absorption of entrapped insulin in normal and diabetic dogs," Biochimica et Biophysica Acta, vol. 716, no. 2, pp. 188-193, 1982.
41. C. Chiang and N. Weiner, "Gastrointestinal uptake of liposomes. II. In vivo studies," International Journal of Pharmaceutics, vol. 40, no. 1-2, pp. 143-150, 1987.
42. A. D. Bangham, M. M. Standish, and J. C. Watkins, "Diffusion of univalent ions across the lamellae of swollen phospholipids," Journal of Molecular Biology, vol. 13, no. 1, pp. 238-252, 1965.
43. L. D. Mayer, P. R. Cullis, and M. B. Bally, "The use of transmembrane pH gradient-driven drug encapsulation in the pharmacodynamic evaluation of liposomal doxorubicin," Journal of Liposome Research, vol. 4, no. 1, pp. 529-553, 1994.
44. D. D. Lasic, "Recent developments in medical applications of liposomes: sterically stabilized liposomes in cancer therapy and gene delivery in vivo," Journal of Controlled Release, vol. 48, no. 2-3, pp. 203-222, 1997.
45. A. S. Ulrich, "Biophysical aspects of using liposomes as delivery vehicles," Bioscience Reports, vol. 22, no. 2, pp. 129-150, 2002.
46. G. Gregoriadis, Liposomes as Drug Carriers. Recent Trend and Progress, John Wiley & Sons, New York, NY, USA, 1988.
47. M. L. Immordino, F. Dosio, and L. Cattel, "Stealth liposomes: review of the basic science, rationale, and clinical applications, existing and potential," International Journal of Nanomedicine, vol. 1, no. 3, pp. 297-315, 2006.
48. R.M. Abra, R. B. Bankert, F. Chen et al., "The next generation of liposome delivery systems: recent experience with tumor-targeted, sterically-stabilized immunoliposomes and active-loading gradients," Journal of Liposome Research, vol. 12, no. 1-2, pp. 1-3, 2002.
49. L. Cattel, M. Ceruti, and F. Dosio, "From conventional to stealth liposomes: a new frontier in cancer chemotherapy," Journal of Chemotherapy, vol. 16, no. 4, pp. 94-97, 2004.
50. P. A. Dinndorf, J. Gootenberg, M. H. Cohen, P. Keegan, and R. Pazdur, "FDA drug approval summary: pegaspargase (Oncaspar) for the first-line treatment of children with acute lymphoblastic leukemia (ALL)," Oncologist, vol. 12, no. 8, pp. 991-998, 2007.
51. R.M.Bukowski, C. Tendler, D. Cutler, E.Rose, M. M. Laughlin, and P. Statkevich, "Treating cancer with PEG intron: pharmacokinetic profile and dosing guidelines for an improved interferon-alpha-2b formulation," Cancer, vol. 95, no. 2, pp. 389-396, 2002.
52. M. W. Fried, M. L. Shiffman, R. K. Reddy et al., "Pegylated (40kDa) interferon alfa-2a (in combination with ribavirin: efficacy and safety results from a phase III, randomized, actively-controlled, multicenter study," Gastroenterology, vol. 120, no. 5, article A55, 2001.
53. G. Molineux, "The design and development of pegfilgrastim (PEG-rmetHuG-CSF, Neulasta)," Current Pharmaceutical Design, vol. 10, no. 11, pp. 1235-1244, 2004.
54. I. C. Macdougall and K. U. Eckardt, "Novel strategies for stimulating erythropoiesis and potential new treatments for anaemia," The Lancet, vol. 368, no. 9539, pp. 947-953, 2006.
55. D. Haussecker, "The business of RNAi therapeutics in 2012," Molecular Therapy-Nucleic Acids, vol. 7, no. 1, article e8, 2012.
56. H. Büning, "Gene therapy enters the pharma market: the short story of a long journey," EMBO Molecular Medicine, vol. 5, no. 1, pp. 1-3, 2013.
57. O. G. Mouritsen and K. Jørgensen, "A new look at lipid-membrane structure in relation to drug research," Pharmaceutical Research, vol. 15, no. 10, pp. 1507-1519, 1998.
58. M. Langner and T. E. Kral, "Liposome-based drug delivery systems," Polish Journal of Pharmacology, vol. 51, no. 3, pp. 211-222, 1999.
59. M. Langner, "Effect of liposome molecular composition on its ability to carry drugs," Polish Journal of Pharmacology, vol. 52, no. 1, pp. 3-14, 2000.
60. D. C. Drummond, M. Zignani, and J. Leroux, "Current status of pH-sensitive liposomes in drug delivery," Progress in Lipid Research, vol. 39, no. 5, pp. 409-460, 2000.
61. I. M. Hafez and P. R. Cullis, "Roles of lipid polymorphism in intracellular delivery," Advanced Drug Delivery Reviews, vol. 47, no. 2-3, pp. 139-148, 2001.
62. R. New, Liposomes - A Practical Approach, vol. 58, IRL Press at Oxford University Press, 1994.
63. R. B. Gennis, Biomembranes:Molecular Structure and Function, Springer, 1989, edited by C. Cantor.
64. K. Ramani, R. D. Miclea, V. S. Purohit, D. E. Mager, R. M. Straubinger, and S.V. Balu-Iyer, "Phosphatidylserine containing liposomes reduce immunogenicity of recombinant human factor VIII (rFVIII) in a murine model of hemophilia A," Journal of Pharmaceutical Sciences, vol. 97, no. 4, pp. 1386-1398, 2008.
65. D. C. Drummond, O. Meyer, K. Hong, D. B. Kirpotin, and D. Papahadjopoulos, "Optimizing liposomes for delivery of chemotherapeutic agents to solid tumors," Pharmacological Reviews, vol. 51, no. 4, pp. 691-744, 1999.
66. V. P. Torchilin and V. S. Trubetskoy, "Which polymers canmake nanoparticulate drug carriers long-circulating?" Advanced Drug Delivery Reviews, vol. 16, no. 2-3, pp. 141-155, 1995.
67. H. Maeda, T. Sawa, and T. Konno, "Mechanism of tumor-targeted delivery of macromolecular drugs, including the EPR effect in solid tumor and clinical overview of the prototype polymeric drug SMANCS," Journal of Controlled Release, vol. 74, no. 1-3, pp. 47-61, 2001.
68. E. Mastrobattista, G. A. Koning, L. Van Bloois, A. C. S. Filipe, W. Jiskoot, and G. Storm, "Functional characterization of an endosome-disruptive peptide and its application in cytosolic delivery of immunoliposome-entrapped proteins," Journal of Biological Chemistry, vol. 277, no. 30, pp. 27135-27143, 2002.
69. G. Bendas, A. Krause, R. Schmidt, and J. Vogel, "Selectins as new targets for immunoliposome-mediated drug delivery: a potential way of anti-inflammatory therapy," Pharmaceutica Acta Helvetiae, vol. 73, no. 1, pp. 19-26, 1998.
70. S. M. Moghimi and J. Szebeni, "Stealth liposomes and long circulating nanoparticles: critical issues in pharmacokinetics, opsonization and protein-binding properties," Progress in Lipid Research, vol. 42, no. 6, pp. 463-478, 2003.
71. K. Maruyama, T. Takizawa, N. Takahashi, T. Tagawa, K. Nagaike, and M. Iwatsuru, "Targeting efficiency of PEG-immunoliposome-conjugated antibodies at PEG terminals," Advanced Drug Delivery Reviews, vol. 24, no. 2-3, pp. 235-242, 1997.
72. V. P. Torchilin, "Recent advances with liposomes as pharmaceutical carriers," Nature Reviews Drug Discovery, vol. 4, no. 2, pp. 145-160, 2005.
73. A. A. Gabizon, "Liposome circulation time and tumor targeting: implications for cancer chemotherapy," Advanced Drug Delivery Reviews, vol. 16, no. 2-3, pp. 285-294, 1995.
74. I. Matsuda, H. Konno, T. Tanaka, and S. Nakamura, "Antimetastatic effect of hepatotropic liposomal adriamycin on human metastatic liver tumors," Surgery Today, vol. 31, no. 5, pp. 414-420, 2001.
75. M. Hashida, M. Nishikawa, F. Yamashita, and Y. Takakura, "Cell-specific delivery of genes with glycosylated carriers," Advanced Drug Delivery Reviews, vol. 52, no. 3, pp. 187-196, 2001.
76. J. H. Felgner, R. Kumar, C. N. Sridhar et al., "Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations," Journal of Biological Chemistry, vol. 269, no. 4, pp. 2550-2561, 1994.
77. H. Farhood, N. Serbina, and L. Huang, "The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer," Biochimica et Biophysica Acta: Biomembranes, vol. 1235, no. 2, pp. 289-295, 1995.
78. D. B. Stanimirovic, M. Markovic, D. V. Micic, M. Spatz, and B. B. Mrsulja, "Liposome-entrapped superoxide dismutase reduces ischemia/reperfusion 'oxidative stress' in gerbil brain," Neurochemical Research, vol. 19, no. 12, pp. 1473-1478, 1994.
79. M. E. M. Cruz, M. M. Gaspar, M. B. F. Martins, and M. L. Corvo, "Liposomal superoxide dismutases and their use in the treatment of experimental arthritis," Methods in Enzymology, vol. 391, pp. 395-413, 2005.
80. Y. Lo, J. Tsai, and J. Kuo, "Liposomes and disaccharides as carriers in spray-dried powder formulations of superoxide dismutase," Journal of Controlled Release, vol. 94, no. 2-3, pp. 259-272, 2004.
81. V. P. Torchilin, Immobilized Enzymes in Medicine, Springer, Berlin, Germany, 1991.
82. M. M. Gaspar, R. Perez-Soler, and M. E. M. Cruz, "Biological characterization of L-asparginase liposomal formulations," Cancer Chemotherapy & Pharmacology, vol. 38, no. 4, pp. 373-377, 1996.
83. J. L. M. Heeremans, R. Prevost, M. E. A. Bekkers et al., "Thrombolytic treatment with tissue-type plasminogen activator (t-PA) containing liposomes in rabbits: a comparison with free t-PA," Thrombosis and Haemostasis, vol. 73, no. 3, pp. 488-494, 1995.
84. M. H. Vingerhoeds, H. J. Haisma, S. O. Belliot, R. H. P. Smit, D. J. A. Crommelin, and G. Storm, "Immunoliposomes as enzyme-carriers (immuno-enzymosomes) for antibody-directed enzyme prodrug therapy (ADEPT): optimization of prodrug activating capacity," Pharmaceutical Research, vol. 13, no. 4, pp. 604-610, 1996.
85. M. J. Fonseca, J. C. Jagtenberg, H. J. Haisma, and G. Storm, "Liposome-mediated targeting of enzymes to cancer cells for site-specific activation of prodrugs: Comparison with the corresponding antibody-enzyme conjugate," Pharmaceutical Research, vol. 20, no. 3, pp. 423-428, 2003.
86. K. W. C. Mok and P. R. Cullis, "Structural and fusogenic properties of cationic liposomes in the presence of plasmid DNA," Biophysical Journal, vol. 73, no. 5, pp. 2534-2545, 1997.
87. A. Hirko, F. Tang, and J. A. Hughes, "Cationic lipid vectors for plasmid DNA delivery," Current Medicinal Chemistry, vol. 10, no. 14, pp. 1185-1193, 2003.
88. B.Martin, M. Sainlos, A. Aissaoui et al., "The design of cationic lipids for gene delivery," Current Pharmaceutical Design, vol. 11, no. 3, pp. 375-394, 2005.
89. K. Lappalainen, I. Jääskeläinen, K. Syrjänen et al., "Comparison of cell proliferation and toxicity assays using two cationic liposomes," Pharmaceutical Research, vol. 11, no. 8, pp. 1127-1131, 1994.
90. M. C. Filion and N. C. Phillips, "Toxicity and immunomodulatory activity of liposomal vectors formulatedwith cationic lipids toward immune effector cells," Biochimica et Biophysica Acta: Biomembranes, vol. 1329, no. 2, pp. 345-356, 1997.
91. P. L. Felgner, T. R. Gadek, M. Holm et al., "Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure," Proceedings of the National Academy of Sciences of the United States of America, vol. 84, no. 21, pp. 7413-7417, 1987.
92. D. C. Litzinger, J. M. Brown, I. Wala et al., "Fate of cationic liposomes and their complex with oligonucleotide in vivo," Biochimica et Biophysica Acta - Biomembranes, vol. 1281, no. 2, pp. 139-149, 1996.
93. D. D. Stuart and T. M. Allen, "A new liposomal formulation for antisense oligodeoxynucleotides with small size, high incorporation efficiency and good stability," Biochimica et Biophysica Acta - Biomembranes, vol. 1463, no. 2, pp. 219-229, 2000.
94. N. Maurer, A. Mori, L. Palmer et al., "Lipid-based systems for the intracellular delivery of genetic drugs," Molecular Membrane Biology, vol. 16, no. 1, pp. 129-140, 1999.
95. S. C. Semple, S. K. Klimuk, T. O. Harasym et al., "Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures," Biochimica et Biophysica Acta: Biomembranes, vol. 1510, no. 1-2, pp. 152-166, 2001.
96. N.Maurer, K. F. Wong, H. Stark et al., "Spontaneous entrapment of polynucleotides upon electrostatic interaction with ethanol-destabilized cationic liposomes," Biophysical Journal, vol. 80,no. 5, pp. 2310-2326, 2001.
97. S. C. Semple, A. Akinc, J. Chen et al., "Rational design of cationic lipids for siRNA delivery," Nature Biotechnology, vol. 28, no. 2, pp. 172-176, 2010.
98. D. D. Stuart, G. Y. Kao, and T. M. Allen, "A novel, long-circulating, and functional liposomal formulation of antisense oligodeoxynucleotides targeted against MDR1," Cancer Gene Therapy, vol. 7, no. 3, pp. 466-475, 2000.
99. A. Akinc, W. Querbes, S. De et al., "Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms," Molecular Therapy, vol. 18, no. 7, pp. 1357-1364, 2010.
100. G. Basha, T. I. Novobrantseva, N. Rosin et al., "Influence of cationic lipid composition on gene silencing properties of lipid nanoparticle formulations of siRNA in antigen-presenting cells," Molecular Therapy, vol. 19, no. 12, pp. 2186-2200, 2011.
101. M. Jayaraman, S. M. Ansell, B. L. Mui et al., "Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo," Angewandte Chemie - International Edition, vol. 51, no. 34, pp. 8529-8533, 2012.
102. J. Milton Harris and R. B. Chess, "Effect of pegylation on pharmaceuticals," Nature Reviews Drug Discovery, vol. 2, no. 3, pp. 214-221, 2003.
103. G. Pasut and F.M. Veronese, "State of the art in PEGylation: the great versatility achieved after forty years of research," Journal of Controlled Release, vol. 161, no. 2, pp. 461-472, 2012.
104. S. Jevševar, M. Kunstelj, and V. G. Porekar, "PEGylation of therapeutic proteins," Biotechnology Journal, vol. 5, no. 1, pp. 113-128, 2010.
105. J. González-Valdez, M. Rito-Palomares, and J. Benavides, "Advances and trends in the design, analysis, and characterization of polymer-protein conjugates for "pEGylaided" bioprocesses," Analytical and Bioanalytical Chemistry, vol. 403, no. 8, pp. 2225-2235, 2012.
106. M. S. Hershfield, "PEG-ADA replacement therapy for adenosine deaminase deficiency: an update alter 8.5 years," Clinical Immunology and Immunopathology, vol. 76, no. 3, part 2, pp. S228-S232, 1995.
107. K. L. Lindsay, C. Trepo, T. Heintges et al., "A randomized, double-blind trial comparing pegylated interferon alfa-2b to interferon alfa-2b as initial treatment for chronic hepatitis C," Hepatology, vol. 34, no. 2, pp. 395-403, 2001.
108. Y. Yamamoto, Y. Tsutsumi, Y. Yoshioka et al., "Site-specific pegylation of a lysine-deficient TNF-α with full bioactivity," Nature Biotechnology, vol. 21, no. 5, pp. 546-552, 2003.
109. R. M. Blaese, K. W. Culver, A. D. Miller et al., "T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years," Science, vol. 270, no. 5235, pp. 475-480, 1995.
110. S. J. Howe, M. R. Mansour, K. Schwarzwaelder et al., "Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients," Journal of Clinical Investigation, vol. 118, no. 9, pp. 3143-3150, 2008.
111. F. Ferrua, I. Brigida, and A. Aiuti, "Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency," Current Opinion in Allergy and Clinical Immunology, vol. 10, no. 6, pp. 551-556, 2010.
112. H.B. Gaspar, A. Aiuti, F. Porta, F. Candotti, M. S. Hershfield, and L. D.Notarangelo, "How I treat ADA deficiency," Blood, vol. 114, no. 17, pp. 3524-3532, 2009.
113. A. Aiuti, F. Cattaneo, S. Galimberti et al., "Gene therapy for immunodeficiency due to adenosine deaminase deficiency," The New England Journal of Medicine, vol. 360, no. 5, pp. 447-458, 2009.
114. S. Ogino, M. L. Gulley, J. T. den Dunnen et al., "Standard mutation nomeclature in molecular diagnostics: practical and educational challenges," Journal of Molecular Diagnostics, vol. 9, no. 1, pp. 1-6, 2007.
115. H. B. Gaspar, "Bone marrow transplantation and alternatives for adenosine deaminase deficiency," Immunology and Allergy Clinics of North America, vol. 30, no. 2, pp. 221-236, 2010.
116. H. Schellekens, W. E. Hennink, and V. Brinks, "The immunogenicity of polyethylene glycol: facts and fiction," Pharmaceutical Research, vol. 30, no. 7, pp. 1729-1734, 2013.
117. M. Noga, D. Edinger, R. Kläger et al., "The effect of molar mass and degree of hydroxyethylation on the controlled shielding and deshielding of hydroxyethyl starch-coated polyplexes," Biomaterials, vol. 34, no. 10, pp. 2530-2538, 2013.
118. A. Besheer, G. Hause, J. Kressler, and K. Mäder, "Hydrophobically modified hydroxyethyl starch: synthesis, characterization, and aqueous self-assembly into nano-sized polymeric micelles and vesicles," Biomacromolecules, vol. 8, no. 2,pp. 359-367, 2007.
119. GMBHFKD, "Hesylation Technology; AAPS National Biotechnology Conference," Toronto, Canada, abstract, 2008.
120. A.O. Elzoghby, W. M. Samy, and N. A. Elgindy, "Albumin-based nanoparticles as potential controlled release drug delivery systems," Journal of Controlled Release, vol. 157, no. 2, pp. 168-182, 2012.
121. E. J. Lee, S. A. Khan, J. K. Park, and K. Lim, "Studies on the characteristics of drug-loaded gelatin nanoparticles prepared by nanoprecipitation," Bioprocess and Biosystems Engineering, vol. 35, no. 1-2, pp. 297-307, 2012.
122. T. Mirshahi, J. M. Irache, C. Nicolas et al., "Adaptive immune responses of legumin nanoparticles," Journal of Drug Targeting, vol. 10, no. 8, pp. 625-631, 2002.
123. L. Vincent, J. Varet, J. Pille et al., "Efficacy of dendrimer-mediated angiostatin and TIMP-2 gene delivery on inhibition of tumor growth and angiogenesis: In vitro and in vivo studies," International Journal of Cancer, vol. 105, no. 3, pp. 419-429, 2003.
124. C. H. Ahn, S. Y. Chae, Y. H. Bae, and S. W. Kim, "Biodegradable poly(ethylenimine) for plasmid DNA delivery," Journal of Controlled Release, vol. 80, no. 1-3, pp. 273-282, 2002.
125. G. F. Paciotti, L. Myer, D. Weinreich et al., "Colloidal gold: a novel nanoparticle vector for tumor directed drug delivery," Drug Delivery, vol. 11, no. 3, pp. 169-183, 2004.
126. G. Kaul and M. Amiji, "Tumor-targeted gene delivery using poly(ethylene glycol)-modified gelatin nanoparticles: in vitro and in Vivo studies," Pharmaceutical Research, vol. 22, no. 6, pp. 951-961, 2005.
127. W. Tan, K. Wang, X. He et al., "Bionanotechnology based on silica nanoparticles," Medicinal Research Reviews, vol. 24, no. 5, pp. 621-638, 2004.
128. C. Kneuer, M. Sameti, U. Bakowsky et al., "A nonviral DNA delivery system based on surface modified silica-nanoparticles can efficiently transfect cells in vitro," Bioconjugate Chemistry, vol. 11, no. 6, pp. 926-932, 2000.
129. C. Kneuer, M. Sameti, E. G. Haltner et al., "Silica nanoparticles modified with aminosilanes as carriers for plasmid DNA," International Journal of Pharmaceutics, vol. 196, no. 2, pp. 257-261, 2000.
130. N. Singh, M. Briones, G. Ott, and D. O'Hagan, "Cationic microparticles: a potent delivery system for DNA vaccines," Proceedings of the National Academy of Sciences of the United States of America, vol. 97, no. 2, pp. 811-816, 2000.
131. M. Junghans, J. Kreuter, and A. Zimmer, "Antisense delivery using protamine-oligonucleotide particles," Nucleic Acids Research, vol. 28, article E45, no. 10, 2000.
132. A. Méndez-Ardoy, K. Urbiola, C. Aranda, C. Ortiz-Mellet, J. M. Garca-Fernndez, and C. Tros de Ilarduya, "Polycationic amphiphilic cyclodextrin-based nanoparticles for therapeutic gene delivery," Nanomedicine, vol. 6, no. 10, pp. 1697-1707, 2011.
133. J. Beloor, C. S. Choi, H. Y. Nam et al., "Arginine-engrafted biodegradable polymer for the systemic delivery of therapeutic siRNA," Biomaterials, vol. 33, no. 5, pp. 1640-1650, 2012.