Chemistry: molecular biology and microbiology – Vector – per se
Reexamination Certificate
2006-02-21
2006-02-21
Priebe, Scott D. (Department: 1635)
Chemistry: molecular biology and microbiology
Vector, per se
C536S023100, C536S023500
Reexamination Certificate
active
07001761
ABSTRACT:
The present invention provides a series of novel dystrophin minigenes that retain the essential biological functions. The expression of the dystrophin minigenes may be controlled by a regulatory element along with a small polyadenylation signal. The entire gene expression cassettes may be readily packaged into a viral vector, preferably an AAV vector. The present invention further defines the minimal functional domains of dystrophin and provides ways to optimize and create new versions of dystrophin minigenes. Finally, the present invention provides a method of treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
REFERENCES:
patent: 4868116 (1989-09-01), Morgan et al.
patent: 5173414 (1992-12-01), Lebkowski et al.
patent: 5399346 (1995-03-01), Anderson et al.
patent: 5449614 (1995-09-01), Danos et al.
patent: 5661033 (1997-08-01), Ho et al.
patent: 5985846 (1999-11-01), Kochanek et al.
patent: 6083750 (2000-07-01), Chamberlain et al.
patent: 6207455 (2001-03-01), Chang
patent: 2003/0216332 (2003-11-01), Chamberlain et al.
patent: 1999318467 (1999-11-01), None
patent: WO 89/06286 (1989-07-01), None
Provisional Application 60/238,848.
T-L Chiu et al., Folding and Design,“ Optimizing energy potentials for success in protein tertiary structure prediction,” May 1998, 3:223-228.
JT Ngo et al., The Protein Folding Problem and Tertiary Structure Prediction, “Computational Complexity, Protein Structure Prediction, and the Levinthal Paradox,” 1994, pp. 433 and 492-495.
Roberts, et.al., Determination of the Exon Structure of the Distal Portion of the Dystrophin Gene by Vectorette PCR. Genomics. 1992, vol. 13, pp. 942-950, especially 947 and 949.
Rosenthal, et.al., Two Human cDNA Molecules Coding for the Duchenne Muscular Dystrophy (DMD) Locus are Highly Homologous. Nucleic Acid Research. 1989, vol. 17, No. 13, p. 5391.
Koenig, et.al., The Complete Sequence of Dystrophin Predicts a Rod-Shaped Cytoskeletal Protein. Cell. Apr. 1998, vol. 53, pp. 219-228, especially pp. 220-221 and 223.
Anderson, W.F. Human Gene Therapy. Apr. 1998, vol. 392, pp. 25-30.
Wang, et.al. Adeno-Associated Virus Vector Carrying Human Minidystrophin Genes Effectively Ameliorates Muscular Dystrophy in mdx Mouse Model. Dec. 2000, vol. 97, No. 25, pp. 13714-13719.
Kunkel, et.al. Analysis of Deletions in DNA From Patients with Becker and Duchenne Muscular Dystrophy. Nature Jul. 1986, vol. 322, No. 3, pp. 73-77.
Watkins, et.al. Immunoelectron Microscopic Localization of Dystrophin in Myofibres. Nature Jun. 1988, vol. 333, No. 30, pp. 863-866.
Koenig, et.al. Detailed Analysis of the Repeat Domain of Dystrophin Reveals Four Potential Hinge Segments That May Confer Flexibility. J. of Biological Chemistry. Mar. 1990, vol. 265, No. 8, pp. 4560-4566.
Monaco, et.al. An Explanation for the Phenotypic Differences Between Patients Bearing Partial Deletions of the DMD Locus. Genomics2, 1988, pp. 90-95.
Hoffman, et.al. Characterization of Dystrophin in Muscle-Biopsy Specimens From Patients with Duchenne's or Becker's Muscular Dystrophy. New England J. Medicine. May 26, 1988, vol. 318, No. 21, pp. 1363-1368.
Bulfield, et.al. X Chromosome-Linked Muscular Dystrophy (mdx) in the Mouse. Proc. Natl. Acad. Sci. USA. Feb. 1984, vol. 81, pp. 1189-1192.
Gussoni, et.al. The Fate of Individual MyoblastsAfter Transplantation Into Muscles of DMD Patients. Nature Medicine. Sep. 1997, vol. 3, No. 9, pp. 970-977.
Barton-Davis, et.al. Aminoglycoside Antibiotics Restore Dystrophin Function to Skeletal Muscles of mdx Mice, J. Clinical Investigation. Aug. 1999, vol. 104, No. 4, pp. 375-381.
Curiel, et.al. Strategies to Adapt Adenoviral Vectors for Targeted Delivery. Annals New York Acad. Sci. 1999, vol. 886, pp. 158-171.
Haj-Ahmad, et.al. Development of a Helper-Independent Human Adenovirus Vector and its Use in the Transfer of the Herpes Simplex Virus Thymidine Kinase Gene. J. of Virology. Jan. 1986, vol. 57, No. 1, pp. 267-273.
Ragot, et.al. Efficient Adenovirus-Mediated Transfer of a Human Minidystrophin Gene to Skeletal Muscle of mdx Mice. Nature. Feb. 18, 1993, vol. 361, pp. 647-650.
Howell, et.al. High-Level Dystrophin Expression after Adeno-Virus Mediated Dystrophin Minigene Transfer to Skeletal Muscle of Dystrophic Dogs: Prolongation of Expression With Immunosuppression. Human Gene Therapy. Mar. 1998, vol. 9, pp. 629-634.
Fink, et.al. Gene Transfer to Neurons Using Herpes Simplex Virus-Based Vectors. Annu. Rev. Neuroscience 1986, pp. 265-287.
Miller, A.Dusty Progress Toward Human Gene Therapy. Blood, J. of the American Soc. of Hematology, vol. 76, No. 2, pp. 271-278.
Naldini, et.al. In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector. Science. Apr. 12, 1996, vol. 272, pp. 263-267.
Flotte, et.al. Gene Expression from Adeno-Associated Virus Vectors in Airway Epithelial Cells. Am J. Respiratory Cell and Molecular Biology. 1992, vol. 7, pp. 349-356.
Samulski, et.al. Helper-Free Stocks of Recombinant Adeno-Associated Viruses: Normal Integration Does Not Require Viral Gene Expression. J. of Virology. Sep. 1989, vol. 63, No. 9, pp. 3822-3828.
Xiao, et.al. Efficient Long-Term Gene Transfer into Muscle Tissue of Immunocompetent Mice by Adeno-Associated Virus Vector. J. of Virology. Nov. 1996, vol. 70, No. 11, pp. 8098-8108.
Kessler, et.al. Gene Delivery to Skeletal Muscle Results in Sustained Expression and Systemic Delivery of a Therapeutic Protein. Proc. Natl. Acad. Sci. USA. Nov. 1996, vol. 93, pp. 14082-14087.
Xiao, et.al. Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy. J. of Virology, Dec. 1998, vol. 72, No. 12, pp. 10222-10226.
Pruchnic, et.al. The Use of Adeno-Associated Virus to Circumvent the Maturation-Dependent Viral Transduction of Muscle Fibers. Human Gene Therapy. Mar. 1, 2000, vol. 11, pp. 521-536.
Greelish, et.al. Stable Restoration fo the Sarcoglycan Complex in Dystrophic Muscle Perfused With Histamine and a Recombinant Adeno-Associated Viral Vector. Nature Medicine. Apr. 1999, vol. 5, No. 4, pp. 439-443.
Song, et.al. Sustained Secretion of Human Alpha-1-Antitrypsin From Murine Muscle Transduced With Adeno-Associated Virus Vectors. Proc. Natl. Acad. Sci. USA. Nov. 1998, vol. 95, pp. 14384-14388.
Kay, et.al. Evidence for Gene Transfer and Expression of Factor IX in Haemophilia B Patients Treated with an AAV Vector. Nature Genetics. Mar. 2000, vol. 24, pp. 257-261.
Chen, et.al. Low-Dose Vaccinia Virus-Mediated Cytokine Gene Therapy of Glioma. J. of Immunotherapy. 2001, pp. 46-57.
Bledsoe, et.al. Cytokine Production in Motor Neurons by Poliovirus Replicon Vector Gene Therapy. Nature Biotechnology. Sep. 18, 2000, vol. 18, pp. 964-969.
Wahlfors, et.al. Evaluation of Recombinant Alphaviruses as Vectors in Gene Therapy. Gene Therapy. 2000, vol. 7, pp. 472-480.
Romano, et.al. Latest Developments in Gene Transfer Technology: Achievements, Perspectives, and Controversies over Therapeutic Applications. Stem Cells. 2000, vol. 18, pp. 19-39.
Lee, , et.al. Lipidic Vector Systems for Gene Transfer. Critical Reviews in Therapeutic Drug Carrier Systems. 1997, vol. 14, No. 2, pp. 173-206.
Zhang, et.al. Long-Term Expression of Human Alpha 1-Antitrypsin Gene in Mouse Liver Achieved by Intravenous Administration of Plasmid DNA Using a Hydrodynamics-Based Procedure. Gene Therapy. 2000, vol. 7, pp. 1344-1349.
Yamashita, et.al. Electroporation-Mediated Interleukin-12 Gene Therapy for Hepatocelular Carcinoma in the Mice Model. Cancer Research. Feb. 1, 2001, vol. 61, pp. 1005-1012.
Acsadi, et.al. Human Dystrophin Expression in mdx Mice After Intramuscular Injection of DNA Constructs. Nature. Aug. 29, 1991. vol. 352, pp. 815-818.
Rando, et.al. Rescue of Dystrophin Expression in mdx Mouse Muscle by RNA/DNA Oligonucleotides. Proc. Natl. Acad. Sci. US.A. May 9, 2000, vol. 97, No. 10, pp. 5363-5368.
Cox, et.al. Dp71 Can Restore the Dystrophin-Associated Glycoprotein Complex in Muscle but Fails to Prevent Dystrophy. Nature Genetics. Dec. 1994, vol. 8, pp. 333-339.
Greenberg, et.al. Exogenous Dp71 Restores the Levels of Dystrophin Associated Proteins but
Asklêpios Biopharmaceutical, Inc.
Fuierer Marianne
Fuierer Tristan
Hultquist Steven J.
Priebe Scott D.
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