Hyperbranched Poly(β-amino ester)s (HPAEs) Structure Optimisation for Enhanced Gene Delivery: Non-Ideal Termination Elimination
Available under a Creative Commons Attribution Non-Commercial Share Alike 4.0 International Licence
3. MEDICAL AND HEALTH SCIENCES
Growing at an impressive pace, gene therapy has emerged as a prospective segment in the biotechnology field and presents a bright outlook, given the increasing investments in R&D and the mounting incidence of genetic disorders . Amid the COVID-19 crisis, the global market for gene therapy, estimated at 1.2 billion dollars in the year 2022, is projected to reach a revised size of 2.7 billion dollars by 2026, according to the Cision PR Newswire. However, the lack of efficient gene delivery vectors still hinders the large-scale manufacturing of gene therapy agents. Viral vectors, such as the adeno-associated virus, lentivirus, retrovirus, and gamma-retrovirus, herpes simplex virus, poxvirus, and vaccinia virus, are the most prevalent gene delivery vectors. Despite their high efficiency, safety concerns, such as severe immune responses and activation of viral components, remain together with a limited payload, compromising their clinical trials [2,3]. Moreover, the high manufacturing cost of viral vectors further impedes their large-scale clinical applications.
Li, Y., He, Z & Lyu, J. (2022). Hyperbranched Poly(β-amino ester)s (HPAEs) Structure Optimisation for Enhanced Gene Delivery: Non-Ideal Termination Elimination. Nanomaterials, vol. 12, pg. 3892. doi:10.3390/nano12213892.