Abstract: Novel methods and compositions are provided for introducing a gene capable of modulating the genotype and phenotype into two or more tissues following systemic administration. The gene can be introduced into a mammalian host by way of an expression vector either as naked DNA or associated with lipid carriers, particularly cationic lipid carriers. Multiple individual tisssues can be transfected using naked DNA. Using a DNA: lipid carrier complex. multiple tissues and cell types can be transfected. The techniques and compositions find use in the palliation or treatment of any of a variety of genetic-based disorders.

Abstract: Methods and compositions for producing a mammal capable of expressing an exogenously supplied gene in cells of the airway are disclosed. Lipid carrier-nucleic acid complexes or nucleic acid abre are prepared then delivered via aerosol or systemically to the lung abre or lung plus extrapulmonary tissues. The invention provides a direct method for transforming pulmonary cells as a means for treating the manifestations of CF in the lung and involved extrapulmonary tissues.

Abstract: Novel methods and compositions are provided for introducing a gene capable of modulating the genotype and phenotype into two or more tissues following systemic administration. The gene can be introduced into a mammalian host by way of an expression vector either as naked DNA or associated with lipid carriers, particularly cationic lipid carriers. Multiple individual tisssues can be transfected using naked DNA. Using a DNA: lipid carrier complex, multiple tissues and cell types can be transfected. The techniques and compositions find use in the palliation or treatment of any of a variety of genetic-based disorders.

Abstract: Methods and compositions are described for the efficient delivery and expression of recombinant polynucleotides in animal cells in vitro or in vivo. In particular, a comprehensive approach of non-viral gene delivery is provided that has been optimized with regards to the vectors delivered, maximizing the amount and duration of gene expression, and methods of conditioning the patient to enhance the efficiency of gene delivery. The described methods find application in both gene therapy, and the functional analysis of cloned gene products in vivo.

Abstract: Compositions comprising lipid carrier-CFTR nucleic acid complexes are disclosed.

Abstract: Methods for the delivery of genes directly to the lungs are disclosed. Liposome-nucleic acid complexes are delivered via aerosol with the subsequent in viva expression of a protein encoded by the delivered gene. The invention provides a convenient method for treating pulmonary disorders, as well as for delivering substances systemically via the lung.

Abstract: A method of preventing clinically manifest Pneumocystis carinii pneumonia in immunocompromised humans is provided wherein an aerosol of pentamidine is administered to the patient in a manner which delivers the aerosol to the patient's alveoli. Also provided is a method of treating Pneumocystis carinii pneumonia in HIV seropositive individuals comprising administering a therapeutically effective amount of pentamidine aerosol to the HIV positive individual through inhalation. In addition, a method is provided of administering pentamidine to a human comprising aerosolizing a pentamidine solution in a nebulizer system having a one-way valve disposed between the nebulizer and the mouthpiece of the nebulizer system.

Abstract: A method of preventing clinically manifest Pneumocystis carinii pneumonia in immunocompromised humans is provided wherein an aerosol of pentamidine is administered to the patient in a manner which delivers the aerosol to the patient's alveoli. Also provided is a method of treating Pneumocystis carinii pneumonia in HIV seropositive individuals comprising administering a therapeutically effective amount of pentamidine aerosol to the HIV positive individual through inhalation. In addition, a method is provided of administering pentamidine to a human comprising aerosolizing a pentamidine solution in a nebulizer system having a one-way valve disposed between the nebulizer and the mouthpiece of the nebulizer system.