Stem cell therapy holds a great deal of promise for correcting a wide range of diseases and degenerative conditions. Stem cells, which are capable of becoming any type of cell within the body, have long been explored because they could potentially be used to create new tissue to repair organs or mend injuries. However, the use of stem cells in the clinic is hampered by several barriers including: 1) difficulty in deriving pluripotent stem cells from the body, 2) low efficiency (fragility) of stem cells when transplanted into sick organs, 3) immune suppression needed to protect transplanted stem cells, and 4) vulnerability to tumor formation in transplanted stem cells. New technologies like CRISPR/Cas9 editing and iPSC technology overcomes each of these barriers, opening new doors for scientists in the field. Today we will discuss how CRISPR/Cas9 gene editing is being applied in order to develop more feasible treatments for degenerative diseases using a patient’s own cells.
Nature’s molecules for healing
The discovery of a family of enzymes that can edit the genome has been hailed as one of the most important breakthroughs in modern biology. Found in bacteria and archaea, these molecular scissors are now used to target and cut DNA at specific locations within the genome. The cell’s own DNA-repair mechanisms then kick in to repair the break, and this process can be used for a variety of purposes: for example, to permanently knock out ‘bad’ genes or insert ‘good’ ones.
The CRISPR‑Cas9 system was adapted from the bacterial immune system, which uses it to protect against viruses by slicing up their DNA. It works by combining two components: an enzyme (Cas9) that cleaves DNA, and a guide molecule (gRNA or sgRNA) that directs it to the right piece of DNA. One advantage of CRISPR–Cas9 over earlier genome-editing tools such as zinc-finger nucleases and TALENs is its simplicity: it is an efficient way to create ‘knockout’ cells, in which genes are permanently switched off.
Adipose Tissue: The Body’s Natural Factory for Multi-Potent Cells
Adipose tissue is a rich source of multipotent cells from which plastic surgery procedures can be performed. For example, fat grafting, also known as autologous fat transfer, which is the transfer of fat from one part of the body, called the donor site, to another part of the body. It is one of the most common procedures that plastic surgeons perform and since there are no allografts or xenografts involved, there is no risk of rejection or disease transmission.
Fat grafting can be used for cosmetic purposes such as facial augmentation and breast reconstruction/augmentation. It has also shown great potential in regenerative medicine with applications in wound healing and arthritis treatment.
The adipose-derived stem cells (ASCs) extracted from adipose tissue have been a driving force behind many modern discoveries in regenerative medicine. Injections of ASCs have shown promising results in treating many degenerative diseases by reducing inflammation and stimulating new growth of tissue.
Recent advances in regenerative medicine
Chemically synthesized RNA-guided Cas9 nuclease (abbreviated as sgRNA) is an artificial RNA-guided endonuclease enzyme that can be programmed to target specific stretches of genetic code. It consists of a single guide RNA and the Cas9 nuclease. This synthetic tool has revolutionized genome editing, allowing scientists to change DNA sequences and modify gene function.
Cas9 was originally discovered in Streptococcus pyogenes, one of the bacteria that cause strep throat. In Streptococcus pyogenes, it plays a role in the bacterial immune system by cutting up foreign DNA. In 2012, researchers realized that this system could be used as a tool for genome editing in other species, including humans.
What is the difference between PRP and Stem Cell Therapy?
The difference between PRP and Stem Cell Therapy is that PRP is used to treat symptoms whereas Stem Cell Therapy is used to treat the cause of the problem. PRP (Platelet Rich Plasma) therapy involves the injection of platelets which contain growth factors that stimulate tissue healing, while Stem Cell Therapy uses mesenchymal stem cells (MSCs) which are capable of becoming multiple types of cells in the body including blood vessels, cartilage, bone, muscle and tendon/ligament. In addition to repairing damaged tissue, MSCs also reduce inflammation and thus help manage pain.
Crispr-Cas9 is a single, remarkably effective treatment that promises to change the field of regenerative medicine. There are still a number of technical details to be worked out and regulations to be ironed out before this particular therapy can start being used in the clinic. However, given the preliminary animal testing results, it is quite possible that this breakthrough could indeed help patients with spinal cord injuries and other forms of paralysis regain movement in as little as two years.