Nanoparticle-mediated gene therapy strategies for mitigating inflammatory bowel disease.
Priyanka VermaAasheesh SrivastavaC V SrikanthAvinash BajajPublished in: Biomaterials science (2021)
Inflammatory bowel disease (IBD) is an autoimmune disorder of the gastrointestinal tract (GIT) where Ulcerative Colitis (UC) displays localized inflammation in the colon, and Crohn's Disease (CD) affects the entire GIT. Failure of current therapies and associated side-effects bring forth serious social, economic, and health challenges. The gut epithelium provides the best target for gene therapy delivery vehicles to combat IBD. Gene therapy involving the use of nucleic acid (NA) therapeutics faces major challenges due to the hydrophilic, negative-charge, and degradable nature of NAs. Recent success in the engineering of biomaterials for gene therapy and their emergence in clinical trials for various diseases is an inspiration for scientists to develop gene therapy vehicles that can be easily targeted to the desired tissues for IBD. Advances in nanotechnology have enabled the formulations of numerous nanoparticles for NA delivery to mitigate IBD that still faces challenges of stability in the GIT, poor therapeutic efficacy, and targetability. This review presents the challenges of gene therapeutics, gastrointestinal barriers, and recent advances in the engineering of nanoparticles for IBD treatment along with future directions for successful translation of nanoparticle-mediated gene therapeutics in clinics.
Keyphrases
- gene therapy
- ulcerative colitis
- clinical trial
- nucleic acid
- healthcare
- small molecule
- mental health
- genome wide
- copy number
- primary care
- public health
- multiple sclerosis
- oxidative stress
- gene expression
- mass spectrometry
- cancer therapy
- dna methylation
- transcription factor
- social media
- high resolution
- replacement therapy
- genome wide analysis