Manufacturing Challenges and Rational Formulation Development for AAV Viral Vectors
نویسندگان
چکیده
منابع مشابه
Viral Vectors for Vaccine Development
Recombinant vectors can be used to deliver antigens and to stimulate immune responses in humans. Viral vectors possess various intrinsic properties which may lend to advantages and disadvantages for usage for a given therapeutic application [reviewed by Larocca and Schlom] [1]. The safety and flexibility of recombinant viral vectors have lead to their usage in gene therapy, virotherapy, and vac...
متن کاملProgress and challenges in viral vector manufacturing.
Promising results in several clinical studies have emphasized the potential of gene therapy to address important medical needs and initiated a surge of investments in drug development and commercialization. This enthusiasm is driven by positive data in clinical trials including gene replacement for Hemophilia B, X-linked Severe Combined Immunodeficiency, Leber's Congenital Amaurosis Type 2 and ...
متن کاملManufacturing of recombinant adeno-associated viral vectors for clinical trials.
The ability to elicit robust and long-term transgene expression in vivo together with minimal immunogenicity and little to no toxicity are only a few features that make recombinant adeno-associated virus (rAAV) vectors ideally suited for many gene therapy applications. Successful preclinical studies have encouraged the use of rAAV for therapeutic gene transfer to patients in the clinical settin...
متن کاملSyngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear. Here we systemically evaluated the biological properties of three types of "empty" ...
متن کاملDesign and construction of functional AAV vectors.
Using the basic principles of molecular biology and laboratory techniques presented in this chapter, researchers should be able to create a wide variety of AAV vectors for both clinical and basic research applications. Basic vector design concepts are covered for both protein coding gene expression and small non-coding RNA gene expression cassettes. AAV plasmid vector backbones (available via A...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Journal of Pharmaceutical Sciences
سال: 2021
ISSN: 0022-3549
DOI: 10.1016/j.xphs.2021.03.024