Optimization strategies and advances in the research and development of AAV‐based gene therapy to deliver large transgenes

Kolesnik, Valeria V.; Nurtdinov, Ruslan F.; Oloruntimehin, Ezekiel Sola; Karabelsky, Alexander V.; Malogolovkin, Alexander S.

doi:10.1002/ctm2.1607

Cited by 3 publications

(1 citation statement)

References 175 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…With immunity proven to play a fundamental role in efficiency of treatment, methods to improve AAV technology to avoid such responses are currently being investigated [ 47 , 48 ]. Strategies addressing immunity, as well as other limitations of AAV, include, but are not limited to, optimizing dosing regimen [ 49 ], further engineering of AAV capsids [ 50 , 51 ], and vector development such as self-complementary AAVs (scAAVs) [ 51 , 52 , 53 , 54 ].…”

Section: Adeno-associated Virus-based Gene Therapymentioning

confidence: 99%

Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases

Sussman,

Liberatore,

Drozdz

2024

Pharmaceutics

View full text Add to dashboard Cite

Gene therapy and its role in the medical field have evolved drastically in recent decades. Studies aim to define DNA-based medicine as well as encourage innovation and the further development of novel approaches. Gene therapy has been established as an alternative approach to treat a variety of diseases. Its range of mechanistic applicability is wide; gene therapy has the capacity to address the symptoms of disease, the body’s ability to fight disease, and in some cases has the ability to cure disease, making it a more attractive intervention than some traditional approaches to treatment (i.e., medicine and surgery). Such versatility also suggests gene therapy has the potential to address a greater number of indications than conventional treatments. Many DNA-based therapies have shown promise in clinical trials, and several have been approved for use in humans. Whereas current treatment regimens for chronic disease often require frequent dosing, DNA-based therapies can produce robust and durable expression of therapeutic genes with fewer treatments. This benefit encourages the application of DNA-based gene therapy to manage chronic diseases, an area where improving efficiency of current treatments is urgent. Here, we provide an overview of two DNA-based gene therapies as well as their delivery methods: adeno associated virus (AAV)-based gene therapy and plasmid DNA (pDNA)-based gene therapy. We will focus on how these therapies have already been utilized to improve treatment of chronic disease, as well as how current literature supports the expansion of these therapies to treat additional chronic indications in the future.

show abstract

Section: Adeno-associated Virus-based Gene Therapymentioning

confidence: 99%

Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases

Sussman,

Liberatore,

Drozdz

2024

Pharmaceutics

View full text Add to dashboard Cite

show abstract

Serotype-agnostic affinity purification of adeno-associated virus (AAV) via peptide-functionalized chromatographic resins

Shastry,

Barbieri,

Minzoni

et al. 2024

Journal of Chromatography A

View full text Add to dashboard Cite

Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives

Chernyi,

Gavrilova,

Saruhanyan

et al. 2024

Biomolecules

View full text Add to dashboard Cite

One of the well-known X-linked genetic disorders is hemophilia, which could be hemophilia A as a result of a mutation in the F8 (factor VIII) gene or hemophilia B as a result of a mutation in the F9 (factor IX) gene, leading to insufficient levels of the proteins essential for blood coagulation cascade. In patients with severe hemophilia, factor VIII or factor IX activities in the blood plasma are considerably low, estimated to be less than 1%. This is responsible for spontaneous or post-traumatic bleeding episodes, or both, leading to disease complications and death. Current treatment of hemophilia relies on the prevention of bleeding, which consists of expensive lifelong replacement infusion therapy of blood plasma clotting factors, their recombinant versions, or therapy with recombinant monoclonal antibodies. Recently emerged gene therapy approaches may be a potential game changer that could reshape the therapeutic outcomes of hemophilia A or B using a one-off vector in vivo delivery and aim to achieve long-term endogenous expression of factor VIII or IX. This review examines both traditional approaches to the treatment of hemophilia and modern methods, primarily focusing on gene therapy, to update knowledge in this area. Recent technological advances and gene therapeutics in the pipeline are critically reviewed and summarized. We consider gene therapy to be the most promising method as it may overcome the problems associated with more traditional treatments, such as the need for constant and expensive infusions and the presence of an immune response to the antibody drugs used to treat hemophilia.

show abstract

Optimization strategies and advances in the research and development of AAV‐based gene therapy to deliver large transgenes

Cited by 3 publications

References 175 publications

Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases

Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases

Serotype-agnostic affinity purification of adeno-associated virus (AAV) via peptide-functionalized chromatographic resins

Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives

Contact Info

Product

Resources

About