Genetically Engineering Stem Cells to Fight Macular Degeneration

Genetically Engineering Stem Cells to Fight Macular Degeneration

Stem cells have been at the forefront of many current medical breakthroughs. Now it looks like scientists are genetically engineering stem cells to fight macular degeneration. It may sound like a sci-fi script but the days of manipulated genetic fears are well behind us. Science has painstakingly studied the many facets of how simple changes to cells can be imprinted and used to significantly treat compromised health. However, up to now, manipulating these cells has been a difficult challenge.

One study, as published by Science News, describes the process,

“Induced pluripotent stem cells (iPS) have the potential to convert into a wide variety of cell types and tissues for drug testing and cell replacement therapies. However, the “recipes” for this conversion are often complicated and difficult to implement.” 

Fortunately, with each study, genetic engineering capabilities continue to advance getting much closer to surgically targeting ways to help the body naturally heal itself. This is good news for those suffering from macular degeneration bringing more hope to the horizon of curing a disease that currently afflicts well over eleven million Americans alone.

Bone Marrow Cells for AMD Treatment

It is not that easy to simply extract a human cell and then re-program it to take the place of a cell that has been compromised by disease. It takes hundreds of trial and error research projects to determine what exact cell can be targeted for genetic engineering. Most of this research has proven minimal in its success as one of the most challenging applications is getting the cells to migrate to the retina

Researchers at the Centre for Genomic Regulation (CRG) in Barcelona have recently discovered a way to use bone marrow cells called chemokine receptors, Ccr5 and Cxcr6, to enhance signaling to the retina. Rodent models have proven that by using these receptors the function of degenerating retinal tissue was preserved. Once the research becomes approved through human trials it can be applied across the mainstream landscape. The procedure will most likely entail extracting bone marrow cells from the patient and re-programming them by packing each full of Ccr5 and Cxcr6 receptors and injecting them either directly into the eye or administering through an arm inoculation for transport to the retina. This means that if macular degeneration is caught early, doctors may soon be able to use Ccr5 and Cxcr6 to stop disease progression and maintain what is left of healthy photoreceptors. For those facing or currently diagnosed with age-related macular degeneration (AMD) this could be a sight saving discovery. 

Lead author and CRG research professor Pia Cosma commented on the process,

 “After the cells are transplanted they need to reach the retina and integrate through its layers, Here we have found a way to enhance this process using stem cells commonly found in the bone marrow, but in principle can be used with any transplanted cells.”

The exciting thing about this study is the discovery of the targeted cells researchers were able to locate. It is almost like finding a needle in a haystack which means there may even be more needles to be detected enabling a whole new approach to eradicating AMD. 

The hope is that after this pioneering exploration, it could become easier for other research teams to correlate specific cell applications to be genetically engineered for not only macular degeneration healing but eradicating other diseases as well.

Genetic Research on the Horizon

As the Barcelona research moves forward, other projects for genetically engineering cells to fight macular degeneration are underway. These are some of the groundbreaking attempts at stopping AMD:

  • Skin Cells

One reason genetic engineering and stem cell research is not attacked by the likes of religious groups and other right wing factions is that it doesn’t need to use cells from an embryo anymore. 

Although embryonic stem cells are the most viable, years ago scientists found a way to extract certain cells from various parts of the body and essentially “wipe them clean” of genetic information. Once this is done, information from the organ that needs new stem cells is “loaded” on to the clean cell and that cell is implanted to hopefully revive dead or diseased cells. 

FierceBiotech reported, 

“Earlier this year, a team led by the National Institutes of Health’s National Eye Institute described a technique for transforming skin cells into rod photoreceptors and then implanting them into mouse models of retinal degeneration.”

  • Müller glia Cells

At the renowned NYC hospital, Mount Sinai, genetic reprogramming of dormant Müller glia (MG) cells in the retina may be able to repair damage done by macular degeneration. MG cells are essential for healthy vision but AMD slowly kills these cells which, up until now, where believed to be unable to sustain restoration. 

In zebrafish, when the MG cells are damaged it has been shown that they are able to self-restore, however in mammals this is not a natural occurrence. Scientists have been able to regenerate the MG cells by first damaging the retina and, upon recovery, surprisingly the body grows new ones. This has been highly controversial as damaging a healthy organ is not the most optimal solution. 

Now, scientists have been able to restore the dormant MG cells through genetic engineering.

The study. ‘Restoration of vision after de novo genesis of rod photoreceptors in mammalian retinas’ which was published in the journal Nature stated that,

“Gene transfer of β-catenin stimulates MG proliferation in the absence of injury in mouse retina. Here we report that following gene transfer of β-catenin, cell-cycle-reactivated MG can be reprogrammed to generate rod photoreceptors by subsequent gene transfer of transcription factors essential for rod cell fate specification and determination…Together, our results provide evidence of vision restoration after de novo [from the beginning] MG-derived genesis of rod photoreceptors in mammalian retinas.”

Genetically engineering stem cells to fight macular degeneration is in full swing and it will only be a matter of time before AMD is finally cured. Although these and many studies like these take several years to show progress, each step gets us a little closer to success.