Models of kidney diseases "in vitro"
The kidney was grown in a Petri dish, and then the genome was edited
Anna Govorova, Infox.ru
Bioengineers from several American universities have managed to make an important step in the development of regenerative medicine, Infox reports.
In a Petri dish of pluripotent stem cells, they grew a real miniature human kidney. Then, with the help of a certain technology, they edited her genome in such a way that she acquired mutations characteristic of two common renal diseases – polycystic kidney disease and glomerulonephritis.
According to the authors, such a miniature kidney can become an excellent model for studying these diseases and for developing new effective methods of their treatment. It is quite possible that with the help of this technology it will be possible to grow kidneys from patients' skin cells, then edit their genome, removing mutations, and implant already healthy organs into the patient's body. It sounds fantastic, but, nevertheless, in the future this technology is likely to become a reality.
Scientists report on their achievement in the latest issue of the journal Nature Communications (Freedman et al., Modeling kidney disease with CRISPR-mutant kidney organoids derived from human).
For the experiment, the authors used human pluripotent stem cells (iPSC) – cells with a unique ability to transform into specialized cells of any organs and tissues.
With the help of certain transcription factors, scientists started the process of their transformation into kidney tissue cells. As a result, a small organoid was formed. Moreover, it was vascularized – that is, it was entwined with a network of tubules and vessels.
Kidney model with a diameter of 1 mm – VM
Such a grown kidney was absolutely healthy, and in order to cause its disease, the authors edited its genome using the so-called CRISPR system. This system is based on the Cas9 protein, which cuts the double-stranded DNA of the cell so that the desired segment can be inserted there. With the help of RNA guides, short guide sequences of RNA, it is possible to show Cas9 the place where it is necessary to perform the incision.
The next stage of the experiment was the implantation of these organoid kidneys with an edited genome into the body of mice, where they successfully took root.
"Now we understand that it is really possible to grow kidney organoids from the patient's own tissues. This is very important, because with this approach, organ rejection during transplantation will not occur. And this is one of the most serious problems in transplantation, along with a shortage of organs. Moreover, our technology shows that we can edit the genome of a diseased organ, turning it into a healthy one," says lead author of the study Benjamin Freedman from Brigham and Women's Hospital in Boston (in a press release from the University of Washington Health Sciences Mini–kidney organoids re-create disease in lab dishes – VM).
Of course, the application of this technology in clinical practice is a matter of the distant future.
In the meantime, the scientist is faced with the task of checking the functionality of such kidneys – that is, whether they will work normally in the body after implantation.
It should be added that recently, for the first time, Japanese bioengineers also managed to grow a real functional kidney from human pluripotent stem cells, which worked perfectly when implanted into the animal's body. However, the authors did not perform any manipulations with her genome at that time.
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30.10.2015