AAV Vectors in Gene Therapy: How Recent Clinical Advances are Unraveling New Potentials?

 


Unraveling Potentials of Adeno-associated virus (AAV) 

Gene therapy research has experienced a resurgence, in part due to the identification and comprehension of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that has generated much interest, particularly in clinical-stage experimental therapeutic procedures. It may be tailored to transfer DNA to target cells. To date, one of the safest gene therapy methods has been the ability to produce recombinant AAV particles free of viral genes but bearing DNA sequences of interest for various therapeutic applications.


Serendipity led to the discovery of the adeno-associated virus (AAV), which was first identified in laboratory adenovirus (AdV) preparations in the mid-1960s, five decades ago. A few research groups began studying the fundamentals of AAV biology out of pure scientific curiosity and without realising its huge potential as a platform for human gene therapy. Over the first 15 to 20 years of AAV research, several crucial aspects of the virus, such as its genomic layout and composition, DNA replication and transcription, pathogenicity, and virion assembly, were described.

Adeno-Associated Virus (AAV) Vector Designs

The most crucial aspect of the logical rAAV vector design is the package size of the expression cassette that will be sandwiched between the two inverted terminal repeats (ITRs). Generally, anything less than 5 kb, including viral ITRs, is sufficient as a starting point. The viral production yields or transgene recombination significantly decrease when rAAV vectors with packaging cassettes longer than 5 kb are attempted (truncations). Large coding sequences, such as complete dystrophin genes, will therefore not fit tightly within AAV vectors.

AAV as a Gene Therapy Vector

Any viral vector needs to be able to adhere to and enter the target cell, effectively transfer to the nucleus, maintain expression in the nucleus, and generally be non-toxic. AAV vectors have successfully met each of these requirements. Several modifications have also enhanced their usability.


Modern AAV vector gene therapy was developed due to several characteristics, chief among them being the absence of pathogenicity and persistence of the wild-type virus. AAV vectors without Rep were created because the Adeno-associated virus (AAV) genome is small and there are worries about Rep's potential effects on cellular gene expression. They also lack the cis-active IEE needed for frequent site-specific integration. The ITRs are kept because packing requires them. As a result, extrachromosomal elements are most frequently seen in modern recombinant Adeno-associated virus (rAAV) vector gene therapy.

Promising AAV Vectors for Gene Therapy in Pipeline

It has been discovered that Adeno-associated virus is a very stable vector, enduring significant temperature and pH changes with little to no activity loss. The concentration at which it is currently being developed and refined, which is around 5 x 1013 particles per milliliter, appears to be the only restriction as of now. This formulation constraint will undoubtedly be overcome soon with the reintroduction of clinical use. However, the remarkable stability of these vectors provides ample opportunity to explore multiple administration paths and particular delivery strategies.


More than 70 businesses are now testing 235+ AAV vectors in the gene therapy landscape. Leading goliaths like BioMarin Pharmaceutical, Gensight Biologics, PTC Therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, UniQure, REGENXBIO, Biogen, Spark Therapeutics (Roche), Sarepta Therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology Medicines, and Ultragenyx Pharmaceutical are currently researching these AAV vector.

What Lies Ahead?

For more than 40 years, researchers have employed gene and other nucleic acid transfer into cells as a research tool. Our better understanding of the genetic factors driving specific diseases has fueled the search for effective gene therapies. Over time, new fields of research have revealed other therapeutic uses for gene delivery. The search for new gene treatment platforms that address these deficiencies has also been fuelled by the limitations of the available small molecule and protein therapy platforms. All of these difficulties are overcome by gene therapies, especially target accessibility. Due to its distinct biology, straightforward structure, and absence of known disease correlations, Adeno-associated virus may be the preferred vector for most gene therapy applications. In practically every clinical setting where it has been used, gene therapy employing rAAV has proven to be secure and well-tolerated. Numerous facets of this vector have been discovered through these studies and basic biology research that could be applied in future projects.

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