Various AAV Serotypes and Their Applications in Gene Therapy: An Overview

Abstract

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Today, many AAV-based gene therapy medications are being investigated in preclinical and clinical trials, and new ones are appearing on the market. In this article, we present a review of AAV discovery, properties, different serotypes, and tropism, and a following detailed explanation of their uses in gene therapy for disease of different organs and systems.

Microfluidic AAV Purity Characterization: New Insights into Serotype and Sample Treatment Variability

“…These limitations are important since 10–13 naturally occurring AAV serotypes and over 100 variants have been purified and studied for gene delivery. ,, The wide range of serotypes and variants makes AAV highly versatile, as they may express different tropisms, or tissue targets, based on their binding receptors (Table ). ,, To further increase their therapeutic robustness, it has been demonstrated that AAV vectors could be equipped with the desired high-affinity ligands, resulting in increased efficiency targeting the desired cells . Having such an extensive repertoire of AAV vehicles is of great clinical significance as it has been reported that over 90% of humans have been infected with AAV, approximately 50% of which may have neutralizing antibodies against the virus. ,,, As such, serotype or variant selection may significantly impact therapy efficiency based on its ability to evade the immune system.…”

“…However, despite the great potential shown by the method, it was limited by (1) using a standard that matched the serotype of the sample, (2) the limited translation to other serotypes due to serotype-specific capsid thermal stability, and (3) the requirement for a time-consuming sample treatment step for nucleic acid extraction. These limitations are important since 10–13 naturally occurring AAV serotypes and over 100 variants have been purified and studied for gene delivery. ,, The wide range of serotypes and variants makes AAV highly versatile, as they may express different tropisms, or tissue targets, based on their binding receptors (Table ). ,, To further increase their therapeutic robustness, it has been demonstrated that AAV vectors could be equipped with the desired high-affinity ligands, resulting in increased efficiency targeting the desired cells .…”

Preparation of Chitosan Nanoparticles through a Readily Solvent-Exchange Process for Efficient and Enhanced Gene Delivery

“…12 However, the persistence of adenovirus gene therapeutics depends on the duration of the therapeutic gene existing in the host cell. 10 Compared with viral vectors, nonviral vectors have the advantages of low immunogenicity, low toxicity, and simple operation. 13 More and more nonviral vectors including cationic polymers, liposomes, polypeptides, etc., have received extensive attention in both academic research and clinical trials.…”

Telomerase-activated Au@DNA nanomachine for targeted chemo-photodynamic synergistic therapy

“…1–3 Although researchers have extensively explored many treatment methods in the past few decades, specific tumour treatment with minimal side effects is still a major challenge. 4 At present, new cancer treatment strategies such as gene therapy 5–9 and immunotherapy 10–13 have been widely studied and used, but chemotherapy 14–20 is still the most commonly used clinical method. One important problem in chemotherapy is the drug resistance of cancer cells, which causes less accumulation of chemotherapeutic drugs in the tumour and makes the effects of the treatment worse.…”