Adeno-Associated Virus-Mediated Gene Therapy.

Choice of vector is the most critical step in gene therapy. Adeno-associated viruses (AAV); third generation vectors, are getting much attention of scientists to be used as vehicles due to their non-pathogenicity, excellent safety profile, low immune responses, great efficiency to transduce non-dividing cells, large capacity to transfer genetic material and long-term expression of genetic payload. AAVs have multiple serotypes and each serotype shows tropism for a specific cell. Different serotypes are used to target liver, lungs, muscles, retina, heart, CNS, kidneys, etc. Furthermore, AAV based gene therapies have tremendous marketing applications that can be perfectly incorporated in the anticipated sites of the host target genome resulting in life long expression of transgenes. Some therapeutic products use AAV vectors that are used to treat lipoprotein lipase deficiency (LPLD) and it is injected intramuscularly, to treat mutated retinal pigment epithelium RPE65 (RPE65) that is introduced to subretinal space, an intravenous infusion to treat spinal muscular atrophy and rAAV2-CFTR vector is introduced into nasal epithelial cells to treat cystic fibrosis. AAV therapies and other such interdisciplinary methodologies can create the miracles for the generation of precision gene therapies for the treatment of most serious and sometimes fatal disorders.

Emerging applications of nanotechnology in context to immunology: A comprehensive review.

Numerous benefits of nanotechnology are available in many scientific domains. In this sense, nanoparticles serve as the fundamental foundation of nanotechnology. Recent developments in nanotechnology have demonstrated that nanoparticles have enormous promise for use in almost every field of life sciences. Nanoscience and nanotechnology use the distinctive characteristics of tiny nanoparticles (NPs) for various purposes in electronics, fabrics, cosmetics, biopharmaceutical industries, and medicines. The exclusive physical, chemical, and biological characteristics of nanoparticles prompt different immune responses in the body. Nanoparticles are believed to have strong potential for the development of advanced adjuvants, cytokines, vaccines, drugs, immunotherapies, and theranostic applications for the treatment of targeted bacterial, fungal, viral, and allergic diseases and removal of the tumor with minimal toxicity as compared to macro and microstructures. This review highlights the medical and non-medical applications with a detailed discussion on enhanced and targeted natural and acquired immunity against pathogens provoked by nanoparticles. The immunological aspects of the nanotechnology field are beyond the scope of this Review. However, we provide updated data that will explore novel theragnostic immunological applications of nanotechnology for better and immediate treatment.