Neglected diseases should not remain so, treat them together
In humans, the interaction of genes with each other and with environmental factors is underlying many aspects of health and disease.
Genetic diseases are usually grouped together in a single gene disorders (hemoglobinopathies, cystic fibrosis and hemophilia) and a chromosomal disorder (trisomy 21, among others). We describe these conditions as genetic diseases because the presence of one or more genes or chromosomes defective leads to the appearance of a pathology.
We speak of a rare disease when a disease affects a small number of people, fewer than one in 2000 in Europe. Rare diseases are counted by thousands, with more than 1000 genes responsible. At the present time, there were in the 7000 genetic diseases, while a further 5 are described each week.
Genetic diseases, hereditary, due to a malfunction of one or several genes. These genetic diseases are linked to a mutation of a gene, a defect that will result in the synthesis of an abnormal protein that, in terms of its importance, will have consequences very different from a genetic disease to the other.
Among the genetic diseases, there are also many of benign or low-disabling (e.g., color blindness) that disorders are extremely serious (for example, cystic fibrosis).
For most rare diseases, there is no curative treatment, but proper care can improve the quality of life and extend the life. Lack of sufficient scientific and medical knowledge, a large number of patients are not diagnosed. Their disease remains unknown. At best, these people are taken care of on the basis of the expression of their symptoms. All patients and their families describe an obstacle course to be listened to, to inform, and to be referred to the competent teams, where they exist, in order to make the right diagnosis.
This site reference, as far as possible, the entirety of the diseases identified by the world Health Organization.
And for tomorrow ?
Our world is changing at high speed, and what seemed impossible yesterday will be commonplace tomorrow. As well, you will find no trouble in processes viable to 100%, allowing you to completely solve your problems chronic acne, your addictions insoluble in the cigarette, the online games or the casino, to the alcohol. Your detox will become all the more easy that tomorrow, probably around the year 2020, the sequencing your DNA has allowed us to demonstrate that your behavioural problems in the face of certain foods (sugar, chocolate... which are also the source of your obesity, and your constant desire to lose weight), your addiction to alcohol, sex, or smoking (although your desire to stop smoking or drinking can be very real), your times of sadness and depression, are the result of some genes that are very specific. Any disease, how it is being genetic in origin, the result will be that all will be healed tomorrow, almost by magic.
You always doubt, because you do not understand a disease, for example, bacterial or viral origin, can be considered to be of genetic origin, in the end, and that is unique to a particular species (and the cross-species transmission is not obvious for most of the diseases). It is important to understand that an individual X, the holder of a genetic heritage X1, will be the prime target of some viruses and some bacteria, while another individual, will never or almost the victim of some virus or bacteria applicants X1. The reason for this ? The genes. The virus looks for the flaws and the entrance doors of your DNA. If you don't, it will remain inactive forever. The bacterium is seeking, for its part, the flaws of your immune system and proliferates if your army of lymphocytes is deficient. However your disability is generally (except as related to environments or supplies that are very unhealthy) has its roots in your genetic heritage. And therefore, your genetics predispose you to certain diseases from the outside, so that your next door neighbor, he is never sick. It's not fool ourselves, even your ability to depress, to be depressed, aggressive, violent, or to think through, is genetic in origin (and the opposite too). That fact that you are a human being, and not an insect or a plant, it is your DNA. And in your DNA are the writings genetic define your strengths and your weaknesses, appearance, intelligence, longevity, and health.
Then, believe it or not, tomorrow, we will find the paths of your healing by correcting your genetic heritage. And this, your living by means of fast, simple, economical and efficient. And this can go very far, since it can as well improve the quality of your diet and digestion (in order to avoid dental repetitive, digestive diseases and others), you offer cures to express against your cancers (e.g. lung cancer related to cigarette smoking, asbestos or toxic products such as paints, solvents...), and this drastic drop in the number of disorders of behavior, and death through chronic diseases, orphan or not, will also improve your living conditions, your longevity, your serenity and your level of life will also be improved as health systems, health insurance and complementary health, will not be more onerous and will display the best rates, and then deliver the appropriate care for all types of diseases.
No. This is not science fiction.
A French company, Cellectis, projects over the past 14 years to solve all kinds of problems similar to those I write about. In the great leader, the company is engaged in the development of patents and inventions, enabling them to develop a engineering of genomes and a variety of tools, such as scissors DNA, the "knock-in" insertion of genes, the "knock-out" inactivation of genes, TALEN, and many others. These tools are able to change in a very secure manner, and a variety of small portions of DNA specific.
Now the company is planning to propose in a future close enough to all sorts of therapies related to the repair of dna sequences that are defective (for example, Duchenne muscular dystrophy could be included among the diseases treated), the contribution of skills to additional genetic or repair of the genome through the elimination of viral sequences such as those of the herpes and HIV.
But in the immediate future, without going so far, the company offers through a project named Sceil to reconstruct from your skin cells, commonly referred to as totipotent stem cells, called IPS, that are not embryonic stem cells (because your embryo has long since disappeared), but transformed cells with similar properties. Thus, Cellectis will be able to provide you with a day of new bodies, or more short-term therapies of stem cells, to repair locally, heart, liver and other organs, muscles and cartilage. And one day in the not too distant future, you may be also freely change the color of your eyes, or change the complexion of your skin, or even change the appearance without any lifting. Of course, these techniques will not be to the point that by 5, 10 or 20 years and more are not yet offered by cellectis and will perhaps never be. But the company offers you already store your skin cells, and to establish a date today of all the changes that will occur tomorrow, because, unlike embryonic stem cells, IPS are aging and, therefore, the more time passes the more your cells will be " aged ". In other words, from a patient 60 years of age, the skin cells will give rise to a later liver brand new, but it will be over the age of 60 years. However, there is no doubt that one day, cellectis will be able to rejuvenate the IPS (by fixing the damaged telomeres) and at that time, another path will open to humanity, that of the rejuvenation genetics. We are not there yet. Cellectis it is also a very large project of allogeneic immunotherapy, which is to heal "total and permanent" almost all cancers through therapies that will be summarized in a single needle for a cost very significantly lower than existing therapies. Cellectis has for objectives to achieve the rate of healing of the order of 100 %. Oncology is very fashionable, and although there are currently several tracks to eradicate the cancer (the starving, the attack by viruses, etc.), it seems to be in the immediate future to be the most plausible in the medium term, the first results on the leukemia, demonstrating the success rate of the order of 89 %.
This is not for now, we still need to wait 2 or 3 years for the first tests successful on the man, but let us rejoice that the current treatments are living their last hours, and that tomorrow we will be able to heal our cancers, such as for treatment of a strong flu. And, incidentally, cellectis is also in the process of developing a whole range of foods non-GMO healthier for your health.
Thus, the world is changing, it is changing faster than we and our certainties unfounded are now a thing of the past. The science-fiction itself belongs to the past. Nanotechnology, biotechnology, information technology and cognitive science (NBIC) are revolutionizing our world and we don't see this yet.
Suffering has its importance in the understanding of good and evil, it brings its values to our thinking and to our conception of the world, but one day the pain itself must cease, because it is only a step in our evolution.