use of liposome as a gene carrier in treatment of

diabetes type 1

ABSTRACT
Liposomes as a gene carrier in the treatment of Diabetes Type 1

Diabetes Type 1 is a chronic autoimmune disease that affects millions of people worldwide
and requires lifelong management. Traditional treatments for Diabetes Type 1, such as
insulin therapy, have limitations and can be associated with complications. Therefore, there
is a need for effective gene therapy in the treatment of Diabetes Type 1[1]. Gastrointestinal
K-cells have been identified as potential target cells in gene therapy for diabetes[2].
Liposomes, which are small spherical vesicles composed of a lipid bilayer, have emerged as
a potential gene carrier for the treatment of Diabetes Type 1. n gene therapy, genetic
materials, such as plasmid DNA, antisense oligonucleotides (asODNs), and small
interfering RNA (siRNA), can be used to treat or prevent disease. This includes replacing a
mutated gene, inactivating a mutated gene, or introducing a new gene. Although gene
therapy is a promising treatment option for a number of diseases (including inherited
disorders, some types of cancer, and certain viral infections), successful gene therapy is
hampered by the lack of effective delivery systems. Liposomal delivery of nucleic acids,
such as plasmid DNA, asODNs, and siRNAs, represents a very promising nanocarrier
system that is relatively safe and effective in delivering genes to targeted locations in the
body. Lipoid-based delivery systems have also been shown to be stable in serum and
plasma, have improved biodistribution, prolonged circulation half-life, and enhanced
target tissue selectivity.

1. Introduction

Liposomes can selectively target specific cells, such as K-cells, while minimizing the risk of off-target
effects[5]. Additionally, liposomes can protect the gene from degradation, prolonging its activity and
enhancing its therapeutic potential[4]. Liposomes can also be designed to be biodegradable and
biocompatible, reducing the risk of adverse reactions[6]. Moreover, liposomes can be tailored to
improve the pharmacokinetics and pharmacodynamics of gene therapy[7].

Several studies have demonstrated the effectiveness of liposomes as a gene carrier in the treatment of
Diabetes Type1.For instance, a study showed that liposomal carriers could deliver siRNA duplexes to
patients' peripheral blood mononuclear cells, leading to a reduction in the expression of the PTPN22
gene[8]. Another study showed that a nano-liposomal carrier system with therapeutic Cas9-RNP had
great potential for the treatment of Type 2 Diabetes Mellitus[1]. Furthermore, a PEGylated liposome
targeted with FA had promising results in decreasing blood glucose levels in vivo[10]. These studies
suggest that liposomes have great potential as a gene carrier in the treatment of Diabetes Type 1, and
further research in this area is warranted.
2. Characteristics

Liposomes are generally formed by the self-assembly of dissolved lipid molecules, each of which
contains a hydrophilic head group and hydrophobic tails. These lipids take on associations which yield
entropically favorable states of low free energy, in some cases forming bimolecular lipid leaflets. Such
leaflets are characterized by hydrophobic hydrocarbon tails facing each other and hydrophilic head
groups facing outward to associate with aqueous solution . At this point, the bilayer formation is still
energetically unfavorable because the hydrophobic parts of the molecules are still in contact with water,
a problem that is overcome through curvature of the forming bilayer membrane upon itself to form a
vesicle with closed edges . This free-energy-driven self-assembly is stable and has been exploited as a
powerful mechanism for engineering liposomes specifically to the needs of a given system.

Lipid molecules used in liposomes are conserved entities with a head group and hydrophobic
hydrocarbon tails connected via a backbone linker such as glycerol Cationic lipids commonly attain a
positive charge through one or more amines present in the polar head group. The presence of positively
charged amines facilitates binding with anions such as those found in DNA. The liposome thus formed is
a result of energetic contributions by Van der Waals forces and electrostatic binding to the DNA which
partially dictates liposome shapes . Because of the polyanionic nature of DNA, cationic (and neutral)
lipids are typically used for gene delivery, while the use of anionic liposomes has been fairly restricted to
the delivery of other therapeutic macromolecules.

The effectiveness of Liposomes as a gene carrier in the treatment of Diabetes Type 1

Liposomes have emerged as a promising gene delivery system for the treatment of various
diseases, including diabetes type 1[4]. Liposomes are spherical vesicles composed of a lipid
bilayer that can encapsulate therapeutic genes and deliver them to target cells[11]. They offer
several advantages as a gene carrier, including their biocompatibility, biodegradability, and
ability to protect the therapeutic genes from degradation[1]. Additionally, liposomes can be
modified to target specific cells, such as pancreatic beta cells, which are crucial in the
pathogenesis of diabetes type 1[10]. These features make liposomes an attractive option for
delivering therapeutic genes to treat diabetes type 1

Studies have demonstrated the effectiveness of liposomes in delivering therapeutic genes for the
treatment of diabetes type 1[12]. In one study, PEGylated liposomes targeted with folic acid
were found to be effective in decreasing blood glucose levels in vivo[10]. Another study found
that pH-sensitive liposomes were more efficient in gene delivery than pH-insensitive
liposomes[13]. Additionally, liposomes have been used to deliver small interfering RNA
(siRNA) duplexes to patients' PBMC, resulting in reduced expression of the C1858T PTPN22
gene[8]. These studies suggest that liposomes can effectively deliver therapeutic genes for the
treatment of diabetes type 1.

Liposomes have several advantages as a gene carrier for the treatment of diabetes type 1,
including their ability to target specific cells and protect therapeutic genes from degradation[14].
Additionally, liposomes can be modified to improve their stability and efficacy, such as by
incorporating pH-sensitive components[15]. Clinical trials have also demonstrated the potential
 of liposomes in treating diabetes type 1, with one study showing that oral insulin delivered using
liposomes was effective in preventing diabetes in relatives of patients with type 1 diabetes[16].
Overall, liposomes offer a safe and efficient gene delivery system for the treatment of diabetes
type 1, with the potential to improve patient outcomes and quality of life[1][10][8].

In conclusion, the use of liposomes as a gene carrier in the treatment of Diabetes Type 1 has
shown great promise. Liposomes have proven to be a safe and efficient gene delivery system,
with the ability to target specific cells. Clinical trials and studies have demonstrated the
effectiveness of liposomes in treating Diabetes Type 1. The advantages of using liposomes over
other gene delivery methods make it a potential game-changer in the treatment of this disease. As
research continues, it is hopeful that liposomes will become a widely used treatment option for
Diabetes Type 1.

References
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1. Drug Delivery System in the Treatment of Diabetes Mellitus. (n.d.) Retrieved December 27,
2023, from www.frontiersin.org/articles/10.3389/fbioe.2020.00880
2. Gene therapy for type 1 diabetes mellitus in rats by .... (n.d.) Retrieved December 27, 2023,
from www.ncbi.nlm.nih.gov/pmc/articles/PMC2725384/
3. Gene therapy in diabetes - PMC. (n.d.) Retrieved December 27, 2023, from
www.ncbi.nlm.nih.gov/pmc/articles/PMC3047781/
4. Barriers to Liposomal Gene Delivery: from Application Site .... (n.d.) Retrieved December
27, 2023, from www.ncbi.nlm.nih.gov/pmc/articles/PMC5242347/
5. Insulin administration via liposomes. (n.d.) Retrieved December 27, 2023, from
pubmed.ncbi.nlm.nih.gov/2226109/
6. Chapter 18 The advantages of liposome-based gene therapy. (n.d.) Retrieved December 27,
2023, from www.ncbi.nlm.nih.gov/pmc/articles/PMC7148944/
7. Updates on Responsive Drug Delivery Based ... - MDPI. (n.d.) Retrieved December 27, 2023,
from www.mdpi.com/1999-4923/14/10/2195
8. Short interfering RNA delivered by cationic liposomes .... (n.d.) Retrieved December 27,
2023, from www.sciencedirect.com/science/article/pii/S1549963418305501
9. Gene liposome nanocomplex-loaded dermal substitute .... (n.d.) Retrieved December 27,
2023, from www.sciencedirect.com/science/article/pii/S0753332223005838
10. Folate targeted PEGylated liposomes for the oral delivery .... (n.d.) Retrieved December 27,
2023, from www.sciencedirect.com
11. The Progress of Non-Viral Materials and Methods for Gene .... (n.d.) Retrieved December
27, 2023, from www.ncbi.nlm.nih.gov/pmc/articles/PMC9695315/
12. Drug Delivery System in the Treatment of Diabetes Mellitus. (n.d.) Retrieved December 27,
2023, from www.ncbi.nlm.nih.gov/pmc/articles/PMC7403527/
13. Liposomes as a gene delivery system. (n.d.) Retrieved December 27, 2023, from
www.scielo.br/j/bjmbr/a/HH67MsTTsqGn6bHWmfYpP6f/
14. Targeted drug delivery strategy: a bridge to the therapy of .... (n.d.) Retrieved December 27,
2023, from www.tandfonline.com/doi/full/10.1080/10717544.2022.2160518
15. Regulatory T cells induced by single peptide liposome .... (n.d.) Retrieved December 27,
2023, from www.ncbi.nlm.nih.gov/pmc/articles/PMC9352518/
16. A Novel Liposome Formulation Carrying Both an Insulin .... (n.d.) Retrieved December 27,
2023, from www.ncbi.nlm.nih.gov/pmc/articles/PMC6855036/