Gene Transfection for Stem Cell Therapy

Abstract

Genetic engineering of stem cells is a strategy that holds promise for realizing the potential therapeutic benefits of stem cell therapy. Through precise control of the stem cell genome, stem cells can replace or repair damaged tissues as well as serve as a depot for the sustained delivery of therapeutic molecules. Various individual genes, genome editing techniques, and transfection agents have been studied and developed for use in stem cell gene transfection. The goal for this review is to introduce specific genes and editing techniques used in stem cell therapy. Diverse gene transfection agents such as liposomes, polymers, dendrimers, peptides, inorganic nanoparticles, and physical transfection agents are also discussed with particular focus on stem cell considerations.

Insight on stem cell preconditioning and instructive biomaterials to enhance cell adhesion, retention, and engraftment for tissue repair

“…There are various strategies currently available for in vitro stem cell manipulation or modification, including physical or chemical manipulation, expansion and culture under prescribed conditions, genetic manipulation, and differentiation before transplantation (Fig. 2, Table 1) [110][111][112]. With the correct combination of techniques, stem cell use may be optimized to reduce the infarct scar, promote cell growth and healing, and augment angiogenesis and neovascularization to improve the functional outcome.…”

“…Various stem cell genetic engineering strategies have been employed to increase survival rate, control differentiation, produce therapeutic proteins, and enhance transplantation efficacy (Fig. 2, Table 1) [6,112,148].…”

“…Some of these challenges are common to many potential therapies and some are unique to this approach. These problems include increasing the efficiency of transfection, controlling gene targeting, limiting the mutagenic potential, and lowering cytotoxicity [112,180,181]. In addition, whatever delivery system is employed must maintain stem cell differentiation status and viability.…”

Recent review of the effect of nanomaterials on stem cells

“…Several adult stem cells, progenitor cells, and induced pluripotent stem cells (iPSCs) have since been isolated and characterized with respect to their potential clinical benet to treat cardiac diseases, supercial wounds, neurologic diseases, and type I diabetes. [54][55][56][57] Transplanted stem cells can rebuild or replace dead tissues and recover existing cells through paracrine effects. However, the stem cell differentiation process in most cases is still necessarily heterogeneous, of which uniformity is critical for preventing tumorigenic potential.…”

“…58 Many research groups have addressed these problems through the development of intracellular delivery of functional molecules, such as DNA, RNAi, peptides, proteins, and drugs that control the stem cell behaviors and fates. 54,59 Some of the drug/chemicals have poor solubility, short halflife and/or poor penetration into cells. Moreover, naked nucleic acids cannot successfully enter cells which require the assistance of a suitable vector.…”