AAV-Mediated Gene Delivery to the Lung

Area of Science:

• * Gene therapy
• * Virology
• * Preclinical research

Background:

• * Adeno-associated virus (AAV) vectors are effective for gene delivery to various tissues, including the lungs.
• * Mouse models are crucial for preliminary preclinical studies of AAV lung gene therapy.
• * Efficient purification and delivery methods are essential for advancing AAV lung gene therapy.

Purpose of the Study:

• * To describe a protocol for purifying adeno-associated virus (AAV) vectors.
• * To outline methods for delivering AAV vectors to the lungs of mice.
• * To support the advancement of AAV lung gene therapy through improved preclinical models.

Main Methods:

• * Purification of AAV vectors utilizing heparin affinity chromatography.
• * Intranasal administration of AAV vectors into mouse lungs.
• * Intratracheal administration of AAV vectors into mouse lungs.

Main Results:

• * A described protocol for AAV purification using heparin affinity chromatography.
• * Established methods for intranasal and intratracheal delivery of AAV vectors in mice.
• * Provided a foundation for further preclinical studies in AAV lung gene therapy.

Conclusions:

• * Heparin affinity chromatography is a viable method for AAV vector purification.
• * Intranasal and intratracheal delivery are effective routes for AAV administration in mouse lungs.
• * These methods facilitate preclinical evaluation of AAV lung gene therapy.