What Is Gene Therapy?

By Petersen Randall / November 27, 2018

Gene therapy is one of those fields of modern medicine that are currently being under the spotlight and high expectation of scientists and patients are focused onto it. The nature gene therapy refers to various methods used to intervene in the genome of a person suffering from some genetic disorder and attempt correction. A wide range of genetic diseases caused by complete missing of a gene, defect structure of a gene or abnormal functioning of a gene, have been incurable so far with only symptomatic therapy available. Gene therapy raises hopes that in relatively near future many groups of patients, even those struggling with disorders causing early death, will be treated with success.

Methods of applying nature gene therapy

Depending on the type of disorder, some genetic diseases are caused by a gene mutation (a small part of the gene is a defect), while others are based on the complete absence of a given gene. Therefore, scientists are developing three possible approaches to gene therapy. The first one refers to gene insertion. The process of multiplication is used to make a sufficient number of copies of a healthy targeted gene, which are then inserted into the body of a patient via various vectors. Defected genes remain inside the body, but inserted genes will produce sufficient amount of healthy and functional proteins. The other gene therapy method refers to gene modification. This method is currently out of our reach due to technological limits, but the basic idea is to conduct direct correction of a defected gene and put it in order. This method would solve all sorts of gene mutations. Gene replacement is the third methods being developed. The therapy would be based on ways to crop-out defect gene and insert healthy, functional copies instead.

Benefits and risks of natural gene therapy

A wide range of potential benefits of nature gene therapy is obvious, although the full capacity and potential of this curing strategy haven’t been explored completely yet. The list of genetic disorders and the spectrum of diseases caused by faulty genes are large and millions of patients suffering from severe conditions would directly benefit from gene therapy. This would prolong their lives, relieve their symptoms and unburden hospitals from regular symptomatic treatments currently provided to these people. On the other hand, there are skeptic people who oppose this treatment stating that it might lead modern medicine to eugenic and enable genetic optimization of people based on non-medical reasons. Simply put, these people are worried about global abuse of this potent treatment which would provide methods for genome interventions which are not medically justified, but ideological agenda.

Other potentials of gene therapy

All previously mentioned methods of applying gene therapy include children or adults and their somatic genes. Aside from this aspect, gene therapy has the potential to modify germ cells and sex genes, causing changes that would be transferred to the next generations. Although this also has medical indications, consequences and side effects are way too blurred and not explored enough at this moment.

Stem Cell Therapy and Treatment – Diseases and Conditions

Do you know what is cell therapy? Namely, the potential of stem cells, multipotent and pluripotent cells, has been actively researched worldwide for years in pretty much all fields of modern medicine. Since these cells have a capacity of developing into any form of more mature and differentiated cells, scientists are testing various experimental stem cell techniques to take advantage of that potential and patent it into effective treatment. Currently, cell therapy is being used for treating blood, skin and eye conditions, but neural stem cell therapy, myocardial stem cell therapy and many more indications are being vividly tested and investigated in clinical trials.

Approved stem cell therapies

Probably the most famous and widely utilized implementation of stem cell therapy is in the hematology. The large scale of blood diseases, including various types of leukemia, anemia, and bone marrow diseases are being successfully treated or at least effectively controlled by blood stem cells. Transplantation of stem cells is also therapy option for many diseases of the immune system. Cancer treatments based on chemotherapy or radiation often cause depletion in the total blood cells to count by damaging marrow bone and blood cells production. This is another indication for stem cell therapy. Cultivation and stimulation of stem cells in specific lab environment allow scientists to create skin grafts, which are used to treat patients with skin defects caused by burns or cancers or various surgical interventions. Finally, ophthalmologists are taking advantage of stem cells as well, since this therapy is used for reparation of the damaged cornea. These fields of stem cells implementation have been proved as efficient, safe and better in cost-benefit ratio compared to the classic treatments available for mentioned conditions.

Experimental stem cell techniques

Along with abovementioned officially approved stem cells therapies, there are numerous techniques and indications currently being clinically tested. All of these therapeutic strategies are considered to be experimental until they pass through every phase of lab and clinical trials successfully. Although the results are yet to be seen, the expectations are high and the effects are rather positive so far. The major medical field that relies a lot on the potential of stem cells therapy is neurology. Application of neural stem cell therapy seems to be a promising approach for treating Parkinson’s disease, Alzheimer’s disease, multiple sclerosis, Huntington’s chorea and spinal cord injuries at any level. Aside from neurology, other branches of medicine are exploring stem cell therapy as well. Myocardial infarction could be treated this way if pluripotent cells are used to create brand-new myocardial tissue. A similar method is being investigated for muscle and tendon injuries, as well as for degenerative conditions of cartilages.

Cord blood stem cells

Sampling blood and stem cells from the umbilical cord after baby’s birth is a well-known procedure and the banks preserving this blood are founded in many countries. Although the number of diseases potentially treatable with these stem cells is somewhat limited, this therapy is still being used due to its great advantage: these cells carry the lowest risk of being rejected by the immune system.

Breakthrough in Advanced Kidney Disease by Stem Cell Genetic Med

High blood pressure and the second type of diabetes, along with atherosclerosis, smoking and high levels of blood sugar and lipids are pandemic health issues of the modern world and all of them may damage kidney tissue and lead to chronic kidney disease. If that happens, two only treatment options medicine offers are dialysis and organ transplant. Hence, there’s an ongoing searching for new treatment options with stem cell kidney transplant being under the spotlight as a potential alternative to currently used treatments.

Specific traits of kidney tissue

Kidneys perform several crucial roles in our body. Their main function is to filtrate urine reabsorbing necessary substances and eliminating toxins and metabolic products. This process regulates mineral, electrolyte and salt balance in the blood. Also, kidneys secrete some essential hormones and have a primary role in maintaining regular blood pressure and blood volume. One of the specific traits of kidneys’ organization is that these organs are built of several different types of cells. Dominant element is nephrons with their system of tubules and channels for excretion. This diversity of cell kidneys are constructed of causes a wide range of kidney diseases emerging from various types of cells. This diversity also makes it difficult for scientists to come up with a uniform treatment for acute or chronic kidney failure, because treatment has to be adjusted according to the type of cells that are the primary cause of the illness.

What can stem cell kidney transplant do?

It’s important to mention that up till now scientists still haven’t found true stem cells in kidney tissue. Several cell types have been identified for some characteristics that are similar to bone marrow stem cells and indicate the potential of these cells to act as stem cells. Currently, two main types of cells are being investigated for their potential to stimulate kidney tissue to naturally regenerates. The first type of cells are so-called mesenchymal stem cells, quite similar to bone marrow stem cells and found around nephrons. The other type of cells refers to induced pluripotent stem cells. These cells are capable of developing into pretty much any type of tissue, the same as embryonic cells act, and this is being used in many labs with a goal to create nephron-like structures transplantable into the regular kidney. Since chronic kidney disease can last for years silently before expressing in dramatic symptoms of kidney failure and forcing the patient into dialysis or transplant, the idea is to use these stem cells to create artificial nephrons and to use them to compensate lost or damaged nephrons and restore kidney function at least to the 50% of normal.

Limitations of stem cell kidney transplant

Currently, the best chances for successful treatment has the strategy of implementing mesenchymal or pluripotent cells to stimulate natural regenerative processes in kidney tissue. However, this approach isn’t effective in patients who have lost more than 70% of their kidney function. They need direct replacement of damaged tissue with a new one, but that procedure is still out of our technical capacity.

Nine Things to Know About Stem Cell Treatments

When disease or trauma damage some organ in our body, ideal treatment would be a complete replacement for the new, healthy tissue. This, however, isn’t possible, but the closest we’ve got to that is with stem cell therapies. This field is constantly developing and stem cell research advancements are being reported every once in a while. Unfortunately, numerous clinics offer stem cell treatments, but the matter is filled with false claims that are not supported scientifically. Thus, here are nine facts to know about stem cell treatments.

Few approved treatments, in spite of stem cell research advancements

Although stem cells researches are being conducted worldwide, the truth is that only a restricted group of diseases is being successfully treated by stem cells therapy. The most utilized is blood stem cell transplantation that restores blood and immune system disorders. Some skin, bone and eye conditions are suitable for this cure as well.

Stem cell therapy is not uniform treatment

There’s no one stem cell treatment that will cure unrelated diseases. There are two types of stem cells in our body: embryonic and tissue related stem cells. The second type remains throughout a lifetime, but has limited potential and is applicable solely to the organ from which it originates.

You can’t just insert stem cell and get a result

Most people have a false impression that stem cells are omnipotent and all it takes is for doctors to implement them at the damaged place and the cells will do the rest. This is false. Scientists use a large number of methods to “teach” stem cells to act in the desired way.

Your stem cells aren’t necessarily safe for you

Theoretically, stem cells originate from your body, but once they’ve been taken out and subjected to dozens of lab manipulations, there are chances that your immune system would reject them after stem cells therapy.

Keep in mind procedure risks

Every medical procedure carries risks, so does this one. The stem cells could come infected, there’s a risk of bleeding after surgery, the risk of additional tissue being damaged while injecting stem cells and finally – the risk of cells starting to grow out of control causing cancer.

Experiments are not clinical trials

Regardless of the fact that even clinical trials are testing the effects of the therapy, these treatments are conducted in a controlled environment under strict medical conditions. This is not equal to subjecting yourself to experimental treatment offered in some clinics.

Stem cell treatment may be for a lifetime

Opposite to widely present belief that once conducted stem cell therapy will restore your damaged tissues, the truth is that stem cells are actually drugs just like any other medicine and are often applied for a lifetime in regular periods.

Check the facts and trust future medicine

Two facts to always keep in mind. Accept only carefully checked and scientifically approved treatments conducted in specialized medical centers. Also, be aware that this field of medicine is rapidly progressing, thus the options you are offered today are not the end of the road for stem cell treatment.


Pros and Cons of Stem Cell Therapy

This relatively new and still insufficiently explored medical field is catching the attention of masses worldwide causing opposite reactions. Some see it as the breakthrough medical treatment with endless therapeutic potential, while others oppose strongly accenting its ethical controversy and all the risks attached to it. The third problem is myriad of misconceptions and false information circling around and people struggling with severe diseases picking these wrong data and hoping for miracles. As with all “magic pills” in medicine, nothing is so simple and the truth is usually somewhere in between opposed extremes. Hence, here are some major pros and cons of stem cells therapy.

Pros of stem cells therapy

Probably the biggest advantage of stem cell therapy is the great regenerative potential of stem cells. Embryonic stem cells, derived from the early stages of embryo development, have an omnipotent capacity of differentiating into all sorts of cells and tissues, but these cells can only be sampled by damaging blastocyst. The other type of stem cells, adult or tissue-specific cells, remain present throughout a lifetime, but their regenerative potential is limited to specific cells of a given tissue. Either way, theoretically, each of these cells has the potential to restore tissue and organs damaged by illness or trauma.

This creates terrain for numerous effective medical treatments which could reverse some severe conditions, restore damaged organ systems or even completely eliminate some genetic disorders. This medical intervention could help patients with cystic fibrosis, diabetes, blood disorders, leukemias, cartilage degeneration, skin damages, neurological diseases. Also, stem cells therapy carries the certain potential of restoring or at least slowing down the aging process.

Cons of stem cells therapy

When talking about the disadvantages of stem cell therapy, it’s important to mention that there are way too many exaggerations. In spite of numerous clinical trials and lab researches ongoing worldwide, only limited number of approved treatments are actually being applied. Other therapeutic potentials of stem cells are still being investigated. This means that stem cells theoretically carry endless potential, but in reality, we’re using only a small share of it. Another downside is a pretty unfamiliar medical field and consequentially unknown side effects of many of these interventions.

Some side effects are detected and explored, but since it’s still a new field, long-term consequences are yet to be explored. Another great downside refers to all ethical dilemmas attached to this medical procedure. The genetic disorder could easily be widely interpreted, thus many people are a sceptic and scared of abusing stem cell therapy under loose criteria to adjust people’s genome for non-medical reasons.

Default risks

As with any other medical intervention, there are some inevitable risks. In order to harvest stem cells as well as in order to implement them back into the body, invasive medical intervention is required. This carries the risk of further tissue damaging, infection, bleeding and allergic reactions. Stem cells changed via lab maneuvering could trigger immune reaction and rejection, as well as lose their growing control and turn to malignant tissue.

How And Why Do Scientists Edit Genes?

By Petersen Randall / September 19, 2018

DNA is considered to be an essence of the life of every living creature since literally all the information required to determine every single trait about a given creature is stored within the DNA sequence. The total amount of DNA material with all the codes and integrated data is usually referred to as genome. Somatic cells contain double-strains of DNA and its amount and characteristics are constant in each cell in one body and the germ cells contain half of that amount. DNA material contained within germ cells is combined among parents and transferred onto the next generations via reproduction. This means that somewhere in your genome is store the code that determines everything: the color of your eyes, the traits of your liver or kidney, as well as some of your mental characteristics.

What is genome editing?

Genome editing is the scientific procedure of modifying DNA sequences by cutting some precisely defined sequences and replacing them, duplicating or deleting them or changing them in any other way. Specific vectors and proteins are used to perform this procedure and indications and implications are many. After sequences are cropped out, integrated repairing mechanisms stitch DNA ends back together and the altered DNA is continuing to regulate biological processes in somewhat desired different manner. The idea of performing and utilizing this strategy isn’t new, but it took decades for science and technology to manage to do it successfully.

The idea was primarily created as a strategy for fighting some genetic diseases and mutations that caused various disorders and defects in the human body, but many other potential utilizations have been recognized over the years. Since DNA regulates everything in our body, including metabolism, energy flow, protein secretion, organ construction, immune response to diseases and myriad of other essential functions, all of which can be distorted and faulty, it was necessary for scientists to decode complete genome and precisely target location of every sequence controlling every function.

How is genome editing performed?

There are several genome editing techniques being developed in many labs worldwide, but the most promising one includes CRISP – Cas9 complex. So far, it’s given the best results, it’s easy to conduct, relatively cheap and safe. Both components are protein complexes capable of altering DNA sequences by recognizing specifically targeted DNA parts and cutting them out. CRISP component functions as the navigation, the guiding system that locates desired genes or inter-genes controlling sequences and Cas9 is an associated protein using lytic reactions to cut DNA at precise spots.

Use of gene editing in medicine

Medicine isn’t the only scientific field that takes advantage of genome editing technology, so do various biotechnology industries and their branches, since it can alter DNA not only in humans but in bacteria, herbs, and animals as well. Medical scientists use it widely to explore the origins of various human traits, to detect primary causes of various diseases and finally to try to intervene directly and reverse numerous conditions by fixing DNA at the spot. This is particularly promising in the field of genetic disorders and heritable diseases.



The True Potential Of Stem Cells

Various ideas and expectation regarding stem cells therapy have been circling in the scientific world for decades, with a lot of hoping and a lot of talking about it. Patients have an even greater expectation, especially those people whose lives depend upon some breakthrough medical approaches that would be capable of treating irreversibly damaged or injured tissues.

What do stem cells do?

Stem cells are fundamental cells out of which every other specifically differentiated cell in our body has originated. Roughly divided, there are two types of stem cells: embryonic stem cells and tissue-related stem cells. The first type is found within pre-implanted embryo form (blastocyst) and has the greatest potential to generate literally any type of defined cells. The second type includes adult stem cells that are tissue-related and located in specific organs till the rest of the life. Their regenerative potential is limited to those specific tissues.

Stem cells are omnipotent, multipotent or sometimes unipotent, but with the ability to generate required traits and transform themselves into cells of the specific tissue with all its morphological and functional characteristics. This means that stem cells represent basic natural material for generation new part of our body. Practical aspects include their utilization in regenerative processes, inducing reparation of damaged tissues and the creation of new, healthy tissue parts to replace the flawed ones.

Strategies behind stem cells

Stem cells transplant is currently the only officially approved medical strategy, although scientists are intensively developing mechanisms to replace damaged tissue with tissue generated out of stem cells instead of inserting the mere cells along with the damaged tissue. Another utilized method is instructing stem cells to induce regenerative processes within the damaged or ill cells of the targeted tissue. Also, numerous labs are investigating details of differentiation processes to determine the exact way stem cells transform into specific tissues. This could lead to artificial creating brand-new organs out of our personal, natural cells.

Officially treatable diseases with stem cells

The most explored, tested and proved stem cell treatment includes blood stem cells transplant used for curing various hematological conditions. Transplantation of bone marrow carries pluripotent blood stem cells into the body and triggers them to start production of healthy and properly functioning blood and immunology cells. This is used to fight various types of leukemia, inborn genetic blood disorder, such as Fanconi anemia, as well as to re-populate bone marrow and bloodstream after some treatments that have damaged it. A similar method is used to regenerate damaged skin areas among patients with burns, scars or skin tumors. Chemically or mechanically damaged superficial layer of the eyes (cornea) is also treatable with stem cells.

Potentially treatable diseases with stem cells

Three main medical fields are exploring potential utilization of stem cells to help their patients. Neurologists are trying to fight neurodegenerative conditions, strokes, and dementia, while endocrinologists hope that stem cells could replace insufficient pancreatic tissue to cure diabetes. Finally, if scientist manages to tech stem cells to trigger regenerative processes, myocardial infarction or any other organ injury could be repaired this way.