Alexey B. Chukhlovin

The ongoing CTT issue concerns different topics of transplantology. A special editorial article written by Boris V. Afanasyev is dedicated to 70-year anniversary of Professor Alexander G. Roumiantsev, an outstanding Russian specialist in pediatric hematology. Under clinical articles, a study by Dr. Tatiana L. Gindina concerns adverse prediction value of complex leukemia cell karyotype. Number of such abnormalities per karyotype, such as stage of disease, proved to be independent prognostic factors in a mixed cohort of Ph+ patients with acute lymphoblastic leukemia at the moment of HSCT. The article combines different additional chromosomal aberrations in acute leukemia and describes them in details.

The next article by Maxim A. Kucher and colleagues presents and substantiates a popular theory on close ties between intestinal microbiota and clinical risks after hematopoietic stem cell transplantation. This hypothesis was inspired by distinct correlations between intestinal dysbiosis and clinical episodes of chronic inflammatory bowel diseases. Assessment of microflora biodiversity and prevalence of distinct bacterial species post-HSCT could be an important tool to assess microbial activation as a factor, e.g., of graft-versushost-disease. The authors present their initial data on fetal microflora transplantation in complicated HSCT cases, aiming to prevent or treat severe aGVHD.

A clinical report by Vadim M. Kemaikin et al. from Astana (Kazakh Republic) shares his experience of bone marrow transplant service at the basis of Astana Center for Urgent Medicine. Six years of development transformed a modest auto-BMT unit to a modern Department for different types of HSCT, including haploidentical grafting with rather good outcomes in the patients. Paroxysmal nocturnal hemoglobinuria (PNH), a clonal blood system membranopathy, is among conditions potentially treatable by BMT. To improve prognosis and quality of life in such patients and other conditions with PNH clone, one may use Eculizumab, a monoclonal antibody against complement component since its infusion may block intravascular complement-mediated RBC hemolysis. In her report, Galina A. Dudina has described an instructive case of PNH in an old patient with MDS, who was successfully treated with Eculizumab, since HSCT approach is not valid at old age, with a number of severe comorbidities.

Under experimental studies we would like to recommend a review by Marina O.Popova and colleagues who discuss potential clinical uses of gene editing for treatment of genetic disorders. The authors concern methodology of the in vitro targeted editing procedure using lentiviral vectors. Next, they delineate a spectrum of inherited monogenic disease which are under clinical trials for gene therapy which contains, e.g., such well-known disorders as primary immune deficiencies, beta-thalassemia etc.

The last short communication reflects a viewpoint by Kristina Khodova, an expert for immunogenetics and development of gene therapy, upon business outlooks of the gene therapy. The author reviews a variety of different approaches to gene therapy, appropriate patent stories, current and prospective investments into the studies.