induced pluripotent stem cells (iPSCs), embryonic stem cells (ESCs)

Oculocutaneous albinism (OCA) is a group of hereditary recessive disorder recognized as a loss of pigmentation. OCA can derive from mutations in different genes that produce melanin. These mutations cause disturbances to get a standard melanin synthesis. There are 7 types of oculocutaneous albinism. These include OCA1, OCA2, OCA3, OCA4, OCA5, OCA6, and OCA7. To help OCA patients, it may include management of such as hats with brims and sunscreens. An effective therapy is unavailable for albinism at present. However, to fight OCA in the future, gene therapy can be used. Gene therapy can include use of such as retrovirus vectors, adenovirus vectors, and CRISPR/Cas9 system. Research results in animal models have shown remarkable advances. It means that the gene therapy will be helpful to treat people with albinism.

A “right to withdraw” from research participation is an important mechanism
for ensuring voluntariness and respecting participant autonomy throughout a study. This right
often is expressed as an absolute, dictating that a participant can withdraw from research
at any time for any reason. However, it is more challenging to define a right to withdraw
when the interactions between a researcher and participant have ended but the researcher is
continuing to use biospecimens provided by the research participant. This issue is becoming
particularly complicated as researchers are generating induced pluripotent stem cell (iPSC)
lines for a broad spectrum of research applications, banking, distribution, commercial product
development, drug screening, and preclinical testing for cell-based therapies. The generation
of iPSCs from biospecimens presents particular challenges for informed consent and the right
to withdraw as researchers develop informed consent documents and as projects undergo ethics
review. Here we consider the reasons why a participant may want to withdraw donated
biospecimens from research, especially when considering the ethical landscape of withdrawal
from iPSC research in particular. We then suggest guidance to help policy makers and ethics
review bodies evaluate the issue of withdrawal in relation to iPSC research.

Proper blood vessel networks are necessary for constructing and re-constructing tissues, promoting
wound healing, and delivering metabolic necessities throughout the body. Conversely, an understanding
of vascular dysfunction has provided insight into the pathogenesis and progression of diseases both
common and rare. Recent advances in stem cell-based regenerative medicine e including advances in
stem cell technologies and related progress in bioscaffold design and complex tissue engineering e have
allowed rapid advances in the field of vascular biology, leading in turn to more advanced modeling of
vascular pathophysiology and improved engineering of vascularized tissue constructs. In this review we
examine recent advances in the field of stem cell-derived vasculature, providing an overview of stem cell
technologies as a source for vascular cell types and then focusing on their use in three primary areas:
studies of vascular development and angiogenesis, improved disease modeling, and the engineering of
vascularized constructs for tissue-level modeling and cell-based therapies.

The discovery of induced pluripotent stem cells (iPSCs) in 2006 has inspired almost all areas of the life sciences. In the first report about iPSCs from Takahashi & Yamanaka, terminally differentiated cells (adult skin fibroblasts)  were reprogrammed into pluripotent cells - very similar to embryonic stem cells (ESCs) - through the ectopic expression of four transcription factors: OCT3/4, SOX2, c-MYC and KFL4. Like ESCs, iPSCs have unlimited capacity to renew themselves; they also can differentiate into any other cell type (e.g. muscle cells, neurons). Since their discovery, an unprecedented high number of research groups have taken up this revolutionising technology, and generated iPSCs from patients with genetic diseases. Patient-specific iPSCs offer a new way to study the diseases on cell types which were not accessible for in vitro studies before: neurons from patients with neurological diseases such as Parkinson’s or Alzheimer’s disease, muscle cells from patients suffering from muscular dystrophy, diseases affecting retina cells, and many more examples. These cells provide an unlimited source of human material for modelling the disease and may facilitate screening for novel drugs. Moreover, using iPSCs for cellular replacement therapy or individualized cell therapy after genetic correction may become a viable option when general obstacles of cellular therapies can be overcome. This review summarizes and comments on approaches that have been taken in the last six years for the study and treatment of human diseases using iPSCs.

Hemophilia A is a hemorrhage disorder inherited according to the X-linked inheritance pattern. It affects about 1 in 4,000 to 10,000 males. Permanent changes in the F8 gene result in hemophilia A. These changes result in an abnormal version of coagulation factor VIII. This abnormal condition determines the kinds of the disorder, namely, severe and mild or moderate hemophilia A. Currently, to treat hemophilia A, infusions of plasma-derived or recombinant factor VIII can be used. However, the cost is extremely high. It makes heavy burdens on the patients, their families or the health care system. Therefore, advanced therapy as iPSCs technique has attracted attention of researchers to research for curing this disorder. For example, to obtain edited DNA segments due to hemophilia A, this technique can be used. Research results in animal models have shown important advances.

This article attempts to elucidate how moral complicity arguments of human induced　pluripotent stem cell (iPSC) research imply for the research in Japan. After the first　chapter describes the background and purpose of this article, the second chapter　introduces three views of moral status of human embryos and their implications for　human embryonic stem cell (ESC) research. The third chapter analyses the characteristics of moral complicity of human iPSC research, especially in terms of promoting wrongdoing and benefiting from wrongdoing. Finally, based on the Japanese views of handling of human embryos and human ESC research, from the viewpoint of consistency, the fourth chapter clarifies which implications moral complicity arguments have for hiPSC research in Japan.

С появлением революционной технологии перепрограммирования соматических клеток в омоложенные стволовые клетки, называемые индуцированными стволовыми клетками (ИПСК),  человечество, казалось бы, получило фонтан вечной молодости и здоровья. Однако врачи не спешат использовать чудо-клетки для исцеления больных, так как  лечение с их помощью оказалось сопряжено с риском заболеть раком. Как же надежно  обезопасить терапию клетками полученными из ИПСК? Этот миниобзор посвящен новейшим разработкам и достижениям в разрешении этой задачи.

Liver transplantation remains the only solution for patients suffering from severe end-stage liver disease. Only 33% of these patients on the transplant waiting list will receive a donation; furthermore, the demand for livers is expected to increase over 20% in the next two decades. Available liver donations are limited now more than ever. Stem cell therapies using human embryonic stem cells (hESCs) and human induced pluripotent stem cells (hiPSCs) are being developed to provide cell therapies in order to bypass orthopedic liver transplantation. Additionally, hESCs and hiPSCs are applied in drug development and screening to help pharmaceuticals manufacture more efficacious drugs and in disease modeling allowing scientists to understand disease progression. Clinical cell therapy trials using hepatocyte transplantation have produced promising initial results. Despite continued advances, the effectiveness of transplanted hepatocytes declines within several months and a liver transplant is still necessary. The current advantage of using cell therapy models is to prolong functional use of a diseased liver until a donor liver is received. The potential use of hiPSCs differentiated into hepatocyte cells (hiPSC-HEPs) can generate an infinite supply of hepatic cells. HiPSCs provide a crucial research platform in the development of more effective protocols for cell therapy, drug development and disease modeling.

Production of human embryonic stem cell (hESC)-derived lung progenitors has broad applicability for drug screening and cell therapy; however, this is complicated by limitations in demarcating phenotypic changes with functional validation of airway cell types. In this paper, we reveal the potential of hESCs to produce multipotent lung progenitors using a combined growth factor and physical culture approach, guided by the use of novel markers LIFRα and NRP1. Lung specification of hESCs was achieved by priming differentiation via matrix-specific support, followed by air-liquid interface to allow generation of lung progenitors capable of in vitro maturation into airway epithelial cell types, resulting in functional characteristics such as secretion of pulmonary surfactant, ciliation, polarization, and acquisition of innate immune activity. This approach provided a robust expansion of lung progenitors, allowing in vivo assessment, which demonstrated that only fully differentiated hESC-derived airway cells were retained in the distal airway, where they aided in physiological recovery in immunocompromised mice receiving airway injury. Our study provides a basis for translational applications of hESCs for lung diseases.

Blood transfusions are routinely done in every medical regimen and a worldwide established collection, processing/storage centers provide their services for the same. There have been extreme global demands for both raising the current collections and supply of safe/adequate blood due to increasingly demanding population. With, various risks remain associated with the donor derived blood, and a number of post collection blood screening and processing methods put extreme constraints on supply system especially in the underdeveloped countries. A logistic approach to manufacture erythrocytes ex-vivo by using modern tissue culture techniques have surfaced in the past few years. There are several reports showing the possibilities of RBCs (and even platelets/neutrophils) expansion under tightly regulated conditions. In fact, ex vivo synthesis of the few units of clinical grade RBCs from a single dose of starting material such as umbilical cord blood (CB) has been well established. Similarly, many different sources are also being explored for the same purpose, such as embryonic stem cells, induced pluripotent stem cells. However, the major concerns remain elusive before the manufacture and clinical use of different blood components may be used to successfully replace the present system of donor derived blood transfusion. The most important factor shall include the large scale of RBCs production from each donated unit within a limited time period and cost of their production, both of these issues need to be handled carefully since many of the recipients among developing countries are unable to pay even for the freely available donor derived blood. Anyways, keeping these issues in mind, present article shall be focused on the possibilities of blood production and their use in the near future.