Targeted retrograde gene delivery for neuronal protection

Drug – bio-affecting and body treating compositions – Whole live micro-organism – cell – or virus containing – Genetically modified micro-organism – cell – or virus

Reexamination Certificate

Rate now

  [ 0.00 ] – not rated yet Voters 0   Comments 0

Details

C435S320100, C435S368000, C435S456000

Reexamination Certificate

active

06998118

ABSTRACT:
Methods are disclosed for transducing neurons with heterologous genes using retrograde viral transport. The methods disclosed employ substantially non-toxic vectors, such as adeno-associated virus vectors, that are capable of retrograde axonal transport to introduce and express genes in the neurons. This method has applications in the mapping of neural pathways, in stimulating or inhibiting the growth of neurons, and in the treatment of various neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis.

REFERENCES:
patent: 4955892 (1990-09-01), Daniloff
patent: 5092871 (1992-03-01), Aebischer et al.
patent: 6632427 (2003-10-01), Finiels et al.
patent: 6800281 (2004-10-01), Aebischer et al.
patent: 2002/0031493 (2002-03-01), Horellou et al.
patent: 2003/0050273 (2003-03-01), Ozawa et al.
Dorlund's Illustrated Medical Dictionary, 28th Edition, W.B. Saunders Co., Philadelphia, PA, 1994, pp. 832, 1131, 1361.
Peterson et al., “Prophylactic neuroprotection of injured entorhinal cortical neurons by retrograde in vivo gene delivery of an anti-apoptotic transgene,” European Journal of Neuroscience 12 (Suppl. 11): 233, Abstract 110.13, Jun. 2000.
Simon et al., “bcl-2 gene therapy exacerbates exitotoxicity,” Human Gene Therapy 10: 1715-1720, Jul. 1, 1999.
Orkin et al., Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy, isssued Dec. 7, 1995 by the US Natl. Inst. of Health, Bethesda, MD.
Verma et al., “Gene therapy—promises, problems and prospects,” Nature 389: 239-242, Sep. 18, 1997.
Rosenberg et al., “Gene therapist, heal thyself,” Science 287: 1751, Mar. 10, 2000.
Hsich et al., “Critical issues in gene therapy for neurologic disease,” Huamn Gene Therapy 13: 579-604, Mar. 20, 2002.
Zou et al., “Prolonged transgene expression mediated by a helper-dependent adenoviral vector (hdAd) in the central nervous system,” Mol. Ther. 20(2): 105-113, Aug. 2000.
Aebischer & Ridet, Recombinant Proteins for Neurodegenerative Diseases: The Delivery Issue, Trends Neurosciences, 2001 24(9):533-40.
Antonawich et al., BCL-2 Transduction, Using a Herpes Simplex Virus Amplicon, Protects Hippocampal Neurons from Transient Global Ischemia, Academic Press, Experimental Neurology 156, 130-137 (1999).
Bartlett et al., Infectious Entry Pathway of Adeno-Associated Virus and Adeno-Associated Virus Vectors, Journal of Virology, Mar. 2000, p. 2777-2785.
Bartlett et al., Selective and Rapid Uptake of Adeno-Associated Virus Type 2 in Brain, Human Gene Therapy 9:1181-1186 (May 20, 1998).
Blomer et al., BCL-XL Protects Adult Septal Cholinergic Neurons from Axotomized Cell Death, Proc. Natl. Acad. Sci. USA vol. 95, pp. 2603-2608, Mar. 1998.
Calamandrei and Alleva, Neuronal Growth Factors, Neurotrophins and Memory Deficiency, Behav Brain Res 1995 Jan 23;66(1-2):129-32.
Carver and Barness, Trophic Factors for the Gastrointestinal Tract, Clin Pernatol (1996) 23(2):265-85.
Cleveland et al., From Charcot to SOD1: Mechanisms of Selective Motor Neuron Death in ALS, Neuron, vol. 24, 515-520, Nov., 1999.
DeFalco et al., Virus-Assisted Mapping of Neural Inputs to a Feeding Center in the Hypothalamus,Science291:2608-2613 (Mar. 2001).
Dolorfo & Amaral, Entorhinal Cortex of the Rat: Topographic Organization of the Cells of Origin of the Perforant Path Projection to the Dentate Gyrus, The Journal of Comparative Neurology 398:25-48 (1998).
Duvoisin, Roger C., Overview of Parkinson's Disease, Annals New York Academy of Sciences,pp. 187-193.
Fawcett J.W., Spinal Cord repair: From Experimental Models to Human Application, Spinal Cord (1998) 36(12):811-7.
Gomez-Isla et al., Profound Loss of Layer II Entorhinal Cortex Neurons Occurs in Very Mild Alzheimer's Disease, The Journal of Neuroscience, Jul. 15, 1996, 16(14):4491-4500.
Gonzalez-Garcia, BXL-X is Expressed in Embryonic and Postnatal Neural Tissues and Functions to Prevent Neuronal Cell Death, Proc. Natl. Acad. Sci, USA, vol. 92, pp. 4304-4308, May 1995.
Hefti et al., Development of Neurotrophic Factor Therapy for Alzeheimer's Disease, Ciba Found Symp. (1996)196:54-69.
Kishi & Cowan, A Quantitative EM Autoradiographic Study of the Commisural and Associational Connections of the Dentate Gyrus in the Rat, Anat. Embryol. 160, 173-186 (1980).
Koliatsos V.E., Biological Therapies for Alzheimer's Disease: Focus on Trophic Factors, Crit Rev Neurobiol (1996) 10(2):205-38.
Latchman & Coffin, Viral Vectors for Gene Therapy in Parkinson's Disease, Rev Neurosci. (2001) 12(1):69-78.
Monahan & Samulski, AA V Vectors: is Clinical Success on the Horizon?, Gene Therapy (2000) 7, 24-30.
Monahan & Samulski, Adeno-Associated Virus Vectors for Gene Therapy: More Pros than Cons?, Mol Med Today. (2000) 6(11):433-40.
Offen et al., Apoptosis as a General Cell Death Pathway in Neurodegenerative Diseases, J Neural Transm Suppl. (2000) 58:153-66.
Peterson et al., Central Neuronal Loss and Behavioral Impairment in Mice Lacking Neurotrophin Receptor p75, The Journal of Comparative Neurology 404:1-20 (1999).
Qing et al., Human Fibroblast Growth Factor Receptor 1 is a Co-Receptor for Infection by Adeno-Associated Virus 2, Nature Medicine, vol. 5, No. 1, pp. 71-77, Jan. 1999.
Senut et. al., Intraneuronal Aggregate Formation and Cell Death after Viral Expression of Expanded Polyglutamine Tracts in the Adult Rat Brain,J Neurosci.Jan. 1, 2000; 0(1):219-229.
Smith-Arica & Bartlett, Gene Therapy: Recombinant Adeno-Associated Virus Vectors, Curr Cardiol Rep. (2001) 3(1):43-9.
Snyder, et al. Effective and Stable Adeno-Associated Virus-Mediated Transduction in the Skeletal Muscle of Adult Immunocompetent Mice,Hum Gene Ther.Nov. 1, 1997; 8(16):1891-900.
Summerford et al., αVβ5 Integrin: a Co-Receptor for Adeno-Associated Virus Type 2 Infection, Nature Medicine, vol. 5, No. 1, pp. 78-82, Jan. 1999.
Terenghi G., Peripheral Nerve Regeneration and Neurotrophic Factors, J Anat (1999) 194 ( Pt 1):1-14.
Yamada et al., Herpes Simplex Virus Vector-Mediated Expression of BCL-2 Prevents 6-Hydroxydopamine-Induced Degeneration of Neurons in the Substantia Nigrain Vivo,Proc. Natl. Acad. Sci., vol. 96, pp. 4078-4083, Mar. 1999.
Yuen EC, The Role of Neurothrophic Factors in Disorders of Peripheral Nerves and Motor Neurons, Phys Med Rehabil Clin N Am. (2001) 12(2):293-306, viii.
Yuen et al., Therapeutic Potential of Neurotrophic Factors for Neurological Disorders, Am. Neurol. (1996) 40(3):346-54.
Xiao et al., Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus, Journal of Virology, Mar. 1998, p. 2224-2232.
Xiao et al., Gene Transfer by Adeno-Associated Virus Vectors into the Central Nervous System, Experimental Neurology 144, 113-124 (1997).
Peel, et al. 2000. Adeno-associated virus vectors: Activity and applications in the CNS.Journal of Neuroscience Methods,98:95-104.
Mellecamps, S. et al. Synaptic Sprouting Increases the Uptake Capacities of Motoneurons in Amyotrophic Lateral Sclerosis Mice. Proced. Nat. Acad. Sci. Jun. 19, 2001, vol. 98, No. 13, pp. 7582-7587.
Chamberlin, N. L. et al. Recombinant Adeno-Associated Virus Vector: Use for Transgene Expression and Anterograde Tract Tracing in the CNS. Brain Research. 1998, vol. 793, pp. 169-175.
Bjorklund, A. et al. Towards a Neuroprotective Gene Therapy for Parkinson's disease: Use of Adenovirus, AAV and Lentivirus Vectors for Gene Transfer of GDNF to the Nigrostriatal System in the Rat Parkinson Model. Brain Research. 2000, vol. 886, pp. 82-98.
Kaplitt, M. G. et al. Long-Term Gene Expression and Phenotypic Correction Using Adeno-Associated Virus Vectors in the Mammalian Brain. Nature Genetics. Oct. 1994, vol. 8, pp. 148-154.
Skorupa, A. F. et al. Sustained Production of Beta-Glucuronidase from Localized Sites After AAV Vector Gene Transfer Results in Widespread Distribution of Enzyme and Reversal of Lysomal Storage Lesions in a Large Volume of Brain in Mucopolysaccharidosis VII Mice. Experimental Neurology. 1999

LandOfFree

Say what you really think

Search LandOfFree.com for the USA inventors and patents. Rate them and share your experience with other people.

Rating

Targeted retrograde gene delivery for neuronal protection does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Targeted retrograde gene delivery for neuronal protection, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Targeted retrograde gene delivery for neuronal protection will most certainly appreciate the feedback.

Rate now

     

Profile ID: LFUS-PAI-O-3694674

  Search
All data on this website is collected from public sources. Our data reflects the most accurate information available at the time of publication.