Journal of Nanomedicine & Biotherapeutic Discovery
Open Access

Nanomedical Gene Therapy Medicine at the Molecular Level

Patrick Hong^{* *}Correspondence: Patrick Hong, Department of Nanomedicine, University of Toronto, `Toronto, Canada, Email:

Author info »

Description

Nano medicine at the intersection of nanotechnology and medicine holds immense promise for revolutionizing healthcare by enabling precise, targeted interventions at the molecular level [1]. Among its many applications, nanomedical gene therapy stands out as a innovative approach for treating genetic disorders and chronic diseases by directly manipulating the expression of specific genes within cells [2]. Nano gene therapy a cutting-edge of nanomedical gene therapy medicine poised to transform the landscape of healthcare by addressing diseases at their root cause with unprecedented precision and efficacy [3].

Nanoparticle delivery system at the heart of nano gene therapy lies a sophisticated nanoparticle delivery system engineered to transport therapeutic genes into target cells with remarkable efficiency and specificity. These nanoparticles, typically composed of biocompatible materials such as lipids or polymers, are designed to encapsulate payloads from degradation in the bloodstream. Surface modifications, such as the attachment of targeting ligands or cell-penetrating peptides, enhance the nanoparticles' ability to navigate through biological barriers and home in on specific cell types, ensuring precise delivery to the desired tissues [4].

Gene editing technologies leverages state-of-the-art gene editing technologies, zinc nucleases, to precisely modify the genetic code within target cells [5]. These technologies enable the correction of disease-causing mutations, the insertion of therapeutic genes, or the modulation of gene expression levels to restore normal cellular function. By harnessing the power of these molecular scissors, Nano gene therapy offers unparalleled precision in editing the human genome, opening new avenues for treating previously incurable genetic disorders.

Targeted therapeutic applications is one of the key advantages of nano gene therapy is its ability to target a wide range of diseases at the molecular level [6]. From monogenic disorders like cystic fibrosis and sickle cell anemia to complex multifactorial diseases such as cancer and cardiovascular disorders, nano gene therapy holds promise for offering personalized therapeutic solutions tailored to each patient's unique genetic makeup. By precisely targeting the underlying molecular mechanisms driving disease progression, nano gene therapy offers the potential for longlasting and curative treatments with minimal off-target effects [7].

Precision medicine and personalized therapy in the era of precision medicine, nano gene therapy represents a paradigm shift towards personalized therapy approaches tailored to individual patients' genetic profiles. Through comprehensive genetic screening and analysis, healthcare providers can identify specific genetic mutations or aberrant gene expression patterns underlying a patient's disease and design customized nano gene therapy formulations to address them. By aligning treatment strategies with each patient's unique molecular signature, nano gene therapy maximizes therapeutic efficacy while minimizing the risk of adverse reactions, ushering in a new era of precision medicine [8].

Safety and ethical considerations while nano gene therapy holds immense promise for revolutionizing healthcare, it also raises important safety and ethical considerations that must be carefully addressed. Ensuring the long-term safety and efficacy of gene therapy interventions, minimizing off-target effects, and safeguarding patient privacy and autonomy are most important concerns. Ethical frameworks and regulatory guidelines must be established to govern the responsible development and deployment of nano gene therapy, balancing the potential benefits with the need to mitigate risks and uphold ethical principles [9].

Nano gene therapy represents a innovative advance in the field of nanomedical gene therapy, offering precise, targeted interventions at the molecular level to treat a wide range of genetic disorders and chronic diseases. By using advanced nanoparticle delivery systems and gene editing technologies, nano gene therapy holds the potential to revolutionize healthcare by offering personalized therapeutic solutions tailored to each patient's unique genetic makeup [10]. The advance of nano gene therapy stands landscape of medicine, offering hope for patients with previously incurable diseases and paving the way towards a future of personalized, precision medicine.

References

1. Yin H, Kanasty RL, Eltoukhy AA, Vegas AJ, Dorkin JR, Anderson DG. Non-viral vectors for gene-based therapy. Nat Rev Genet. 2014;15(8):541-555.

   [Crossref] [Google Scholar] [PubMed]

2. Wang Y, Huang L. A window onto siRNA delivery. Nat Biotechnol. 2013;31(7):611-612.

   [Crossref] [Google Scholar] [PubMed]

3. Akinc A, Maier MA, Manoharan M, Fitzgerald K, Jayaraman M, Barros S, et al. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat Nanotechnol. 2019;14(12):1084-1087.

   [Crossref] [Google Scholar] [PubMed]

4. Davis ME, Zuckerman JE, Choi CH, Seligson D, Tolcher A, Alabi CA, et al. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nat. 2010;464(7291):1067-1070.

   [Crossref] [Google Scholar] [PubMed]

5. Maeda H, Nakamura H, Fang J. The EPR effect for macromolecular drug delivery to solid tumors: Improvement of tumor uptake, lowering of systemic toxicity, and distinct tumor imaging in vivo. Adv Drug Deliv Rev. 2013;65(1):71-79.

   [Crossref] [Google Scholar] [PubMed]

6. Mingozzi F, High KA. Immune responses to AAV vectors: Overcoming barriers to successful gene therapy. Blood. 2013;122(1):23-36.

   [Crossref] [Google Scholar] [PubMed]

7. Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex genome engineering using CRISPR/Cas systems. Sci. 2013;339(6):819-823.

   [Crossref] [Google Scholar] [PubMed]

8. Wang HX, Song Z, Lao YH, Xu X, Gong J, Cheng D, et al. Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide. Proceed Nat Acad Sci. 2018;115(19):4903-4908.

   [Crossref] [Google Scholar] [PubMed]

9. Zhao Y, Huang L. Lipid nanoparticles for gene delivery. Adv Genet. 2014;88:13-36.

   [Crossref] [Google Scholar] [PubMed]

10. DeRosa F, Guild B, Karve S, Smith L, Love K, Dorkin JR, et al. Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system. Gene Ther. 2016;23(10):699-707.

    [Crossref] [Google Scholar] [PubMed]