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Can we use gene therapy to fight the skin disorder epidermolysis bullosa?

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On March 29, 2024

In chemical basis of life, integumentary system, tissues

Teachable moment in classrooms:

  1. cellular basis of life chapter – concept of one gene, one protein
  2. cellular basis of life chapter – concept of gene mutation leading to protein malfunction
  3. tissue chapter – the basement membrane is a thin layer of tissue in-between epithelium and the underlying connective tissue
  4. integumentary system chapter –the integrity of the skin depends on the secure connection of the dermis to the basement membrane

The news item:  Recently the following news item appeared online:

U.S. FDA approves Krystal Biotech’s skin-disorder gene therapy

(Reuters) -The U.S. Food and Drug Administration on Friday approved Krystal Biotech Inc’s first-of-its kind topical gene therapy for patients with a genetic skin disorder, sending its shares up 7% in afternoon trading. Patients with the rare dystrophic epidermolysis bullosa disorder suffer from open wounds, causing skin infections and are at an increased risk of vision loss, scarring and skin cancer.

The article states that the therapy named Vyjuvek healed skin wounds in 65% of dystrophic epidermolysis bullosa patients as opposed to 22% healing rate with placebo treatment. The article also states that about 10,000 people world-wide, and 3000 people in the USA suffer from the disorder.

So, Why Do I Care??  While severe forms of epidermolysis bullosa are life-threatening, moderate forms are devastating to the quality of life, as they it creates painful wounds or blisters on the skin, or in the mouth, and the healing wounds lead to widespread scarring. That makes dressing into clothes, wearing shoes, eating difficult. For dystrophic epidermolysis bullosa patients the promise is the possibility of returning to a normal lifestyle after this new treatment.

Plain English, Please!!!  First, let’s talk about epidermolysis bullosa. This is a group of diseases with the shared symptoms of fragile, easy to break skin and blistering of the skin. The three-layered area of epithelium-basement membrane-dermis is made sturdy by several molecules, and if one is malfunctioning because of a mutation, then fragility of the skin results. The many types of epidermolysis bullosa are caused by a variety of genetic mutations.

Second, let’s talk about the cause of dystrophic epidermolysis bullosa. In this type of the disease the missing component is type VII collagen, the molecule that secures the basement membrane to the dermis. Imagine a large picnic table where the table cover is clamped to the table so the wind wouldn’t blow the cover away. Type VII collagen acts like those clamps securing the dermis (the table) to the table cover (the basement membrane). When the type VII college gene suffers mutations, the resulting type VII collagen protein clamp has a distorted shape, and cannot secure the basement membrane to the dermis, and the skin blisters to form open wounds.

Third, let’s talk about how Vyjuvek treatment works. This treatment is delivering a normal type VII collagen gene to the fibroblast cells of the dermis. The gene is enclosed in an inactivated virus, and the viruses are mixed into a gel, and that gel is applied to the wound surface.  Once that virus enters inside the fibroblast in the person’s dermis, then the normal gene is used to make the type VII collagen protein. With time the newly-made, normal type VII collagen “clamps” will secure the dermis to the basement membrane, and prevent new blistering or breaking of the skin.

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Can we really grow blood in a laboratory?

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On January 28, 2024

In cardiovascular system, cellular basis of life, chemical basis of life

Teachable moment in classrooms:

  1. cellular basis of life chapter – concept of one gene, one protein
  2. cellular basis of life chapter – concept of gene mutation leading to protein malfunction
  3. cardiovascular system chapter – formation of red blood cells by stem cells in red bone marrow
  4. respiratory system chapter – oxygen transport by hemoglobin in red blood cells

The news item:  Recently the following report appeared online:

Lab-grown blood given to people in world-first clinical trial

It is hoped the blood could revolutionise care for people who need regular donations.

The report described the need for blood transfusion for repeated infusions such as those for sickle cell anemia patients, and the shortage of minor blood types for transfusion. The report briefly described the lifespan of the red blood cells, and the process of growing blood in the lab.

So, Why Do I Care??  Blood transfusion, the administering of blood or red blood cells into the vein of the recipient, is a life saver when the recipient lost lot of blood, or when the red blood cells of the recipient are being damaged because of a disease. As the transfused blood is derived from blood donors who are not always available to donate, the volume of donated blood can be so low as to create a blood shortage.  It would be a great improvement if red blood cells could be “manufactured” on demand.  That would make blood shortages obsolete. In the US 100,000 people suffer from sickle cell anemia.

Plain English, Please!!!  First, let’s talk about why blood transfusion is a lifesaver clinical intervention, in general. We need red blood cells to transport oxygen from the lungs to all organs of our body. When blood loss or damage to red blood cells (like in sickle cell anemia) decrease the number of oxygen transporter cells, the organs do not receive enough oxygen for normal functioning. It’s like a fleet of trucks delivering bread to a store. If the trucks break down, the bread never get’s to the stores, and the functioning of the store will suffer. In this case the red blood cell trucks deliver oxygen to organs of the body. The most noticeable effect of oxygen deficit is on the skeletal muscles (loss of muscle strength) and on the nervous system (fatigue, tiredness). Restoring the number of red blood cells to normal helps to return the functioning of the body to normal. This why most transfusions do not transfuse whole blood, but only red blood cells.

Second, let’s talk about why sickle cell anemia patients need frequent blood transfusions. In sickle cell anemia patients the mutation of the globin protein of hemoglobin causes the red blood cells to change

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Can astronauts prevent bone loss by eating genetically engineered lettuce?

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On November 27, 2022

In chemical basis of life, digestive system, endocrine system, skeletal system

TeachableMedicalNews article 11272022

Teachable moment in classrooms:

  1. cellular basis of life chapter – concept of one gene, one protein
  2. skeletal system chapter – actions of osteoblasts and osteoclasts in living bones
  3. skeletal system chapter – osteons and trabeculae provide structural strength to bones
  4. endocrine system chapter – actions of PTH made by parathyroid gland
  5. digestive system chapter – ingested proteins are degraded by several digestive enzymes

 

The news item:  Recently the following report appeared:

Humans on Mars may feast on gene-edited salad to stop bones breaking

Genetically modified salad could be crucial to keeping the bones of humans on Mars healthy.

 

 

The article states that a human gene was added to lettuce, and that the human protein, parathyroid hormone (PTH), will fight the osteoporosis that astronauts develop in space. Astronauts lose 1.5% of bone mass from weight-bearing bones, and the expectation is the PTH made by the lettuce will prevent bone loss on a trip to Mars that may last for over a year.

So, Why Do I Care??  Osteoporosis, as stated in the article, weakens bones, but not only in astronauts. Over 10 million people in the US alone has osteoporosis that makes fractures more likely. PTH is currently administered through injections, so, if we could demonstrate that eating PTH-enriched lettuce, then treatment of osteoporosis may become easier.

Plain English, Please!!! First, let’s talk about what osteoporosis is. In our bones we need structural reinforcement to keep bones from fracturing when we walk, run, jump or fall. The microscopic building units called osteons and trabeculae are the structural elements that provide such reinforcement.  Imagine a tall building or a bridge; columns or pillars are the elements of structural reinforcement in them. A corrosion of those pillars and columns weakens the building, and may cause their collapse. In our bones the cells called osteoclasts erode the osteons and trabeculae, but the cells called osteoblasts repair, and, thus, reverse that erosion. In old age, or with hormonal changes, such as menopause, the osteoblasts slow down, and the osteoclasts erode osteons and trabeculae. Osteoporosis is the condition where the erosion weakens the entire bone, and fractures are more likely.

Second, let’s talk about why weightlessness leads to osteoporosis. When we move around in regular gravity, our bone cells are under pressure caused by the weight of the body. That pressure makes

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Can a new pharmaceutical lower blood cholesterol levels to improve cardiovascular health?

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On October 30, 2021

In cardiovascular system, cellular basis of life, chemical basis of life, digestive system

TeachableMedicalNews article 10312021

Teachable moment in classrooms:

  1. chemical basis of life chapter – cholesterol is a steroid type lipid
  2. cellular basis of life chapter – cholesterol is needed to stabilize plasma membranes of our cells
  3. digestive system chapter – liver synthesizes cholesterol
  4. cardiovascular system chapter – coronary artery disease starts with cholesterol buildup in the arteries

The news item:  Recently a report appeared about lowering our blood cholesterol level:

 

FDA approves drug that lowers cholesterol in a new way

Nexletol was approved for people genetically predisposed to have sky-high cholesterol and people who have heart disease and need to further lower their bad cholesterol.

The article stated that high LDL levels are connected with risk of heart attacks, and that this new drug blocks the cholesterol making enzyme in the liver. This new drug, Nexletol, acts differently from the statin drugs that also lower blood cholesterol.

So, Why Do I Care??  In the USA over 1 million people die yearly from heart attacks and strokes, and those deaths are at least partially caused by high blood cholesterol causing blockage inside the blood vessels. While there are life style modifications and drugs we can use to lower blood cholesterol, new pharmaceuticals may increase the success of lowering blood cholesterol.

Plain English, Please!!!   First, let’s talk about what cholesterol is and how it causes cardiovascular disease. Cholesterol is an organic molecule classified as a steroid type lipid. Cholesterol makes cell membranes sturdy, so all cells need it, and cholesterol transporting trucks, called LDL (low density

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Can a genetic mutation of cancer cells be exploited to kill them?

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On March 28, 2021

In cellular basis of life, chemical basis of life, digestive system, reproductive system

TeachableMedicalNews article 03282021

Teachable moment in classrooms:

  1. chemical basis of life chapter – DNA structure as double helix
  2. cellular basis of life chapter – DNA replication during mitosis
  3. cellular basis of life chapter – DNA mutation of a gene usually diminishes protein function
  4. digestive system – anatomy of pancreatic duct (origin of most pancreatic cancers)
  5. reproductive system – anatomy of ovary (surface epithelium is the origin of most ovarian cancers)

The news item:  Recently this article appeared online about a new pancreatic cancer treatment:

FDA approves drug for treating pancreatic cancer

Health regulators clear AstraZeneca and Merck treatment for disease projected to kill 46,000 Americans this year.

The article states that Lynparza has been approved for the treatment of ovarian cancers with BRCA mutation, and this approval is now extended to pancreatic cancers with the same mutation. The article also states that the BRCA mutation hinders fixing DNA damage, that Lynparza is a PARP inhibitor, and that 46,000 Americans die yearly because of pancreatic cancer.

So, Why Do I Care??  Pancreatic cancer has a very low survival rate, because the diagnosis usually happens in the late stage of the cancer. A new drug will likely increase survival rates among those 46,000 patients.

Plain English, Please!!!   First, let’s talk about the BRCA mutation. When cells undergo mitosis (cell division) the DNA is copied (replicated), so the new cell can have as much DNA as the starter cell.

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How does a new antibiotic called Xenleta fight bacterial infections?

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On October 9, 2020

In cellular basis of life, chemical basis of life, lymphatic and immune system, respiratory system

TeachableMedicalNews article 10092020

Teachable moment in classrooms:

  1. chemical basis of life chapter – proteins are made of amino acids linked by peptide bonds
  2. cellular basis of life chapter – protein synthesis on ribosome
  3. respiratory system chapter – alveoli location and function
  4. immune system chapter – events during inflammation
  5. microbiology – bacterial resistance against antibiotics

The news item:  Recently the news of a novel antibiotic was reported:

FDA approves new antibiotic for bacterial pneumonia

A new antibiotic has been approved by the Food and Drug Administration. It’s called Xenleta and the FDA says it’s another treatment option to fight a common type of pneumonia.

The article states that the new antibiotic, Xenleta, is a pleuromutilin, and it fights community acquired bacterial pneumonia.

So, Why Do I Care??

Pneumonia is the reason for 1 million hospitalizations and 50,000 deaths each year in the US. Most pneumonia cases are caused by bacterial infections. Because this disease can get worse quickly, efficient treatment must include the killing of the bacteria that cause the disease. Any new antibiotic that can treat people whose pneumonia resists the regular antibiotics likely to save thousands of lives each year.

Plain English, Please!!!

First, let’s talk about what bacterial pneumonia is. When the deepest portion of our respiratory system, the alveoli (air sacks), are attacked by bacteria, the result is local

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What makes some hand sanitizers dangerously toxic?

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On August 28, 2020

In cellular basis of life, chemical basis of life, digestive system, metabolism, nervous system, Uncategorized

TeachableMedicalNews article 08282020

Teachable moment in classrooms:

  1. chemical basis of life chapter – adenosine triphosphate (ATP)
  2. cellular basis of life chapter –sites of detoxification inside the cell
  3. cellular basis of life chapter – mitochondria as site of ATP production
  4. digestive system chapter – absorption through stomach mucosa
  5. metabolism chapter – electron transport chain as most efficient ATP producing process inside mitochondria
  6. special senses chapter – gross anatomy of optic nerve

The news item:  A recent news item described over a hundred hand sanitizer brands are being removed from store shelves because they may be dangerously toxic:

FDA says to avoid these 130 hand sanitizer products that may not work, or are toxic

Regulators say some of the products contain dangerous levels of methanol, which can lead to blindness, hospitalization and even death.

The article mentions that the toxic ingredient is methanol, and that it may cause blindness.

So, Why Do I Care??  Poisoning by methanol, also called wood alcohol, sickens over 20,000 people each year in the US.  These people may become blind in 24 hours, and suffer long-lasting damage to their kidneys and brain. The best prevention is not to consume methanol either by drinking or through contact with skin, such as through hand sanitizers.  So, taking hand sanitizers off the shelf is part of the prevention.

Plain English, Please!!! First, let’s talk about what methanol is. Methanol is chemically similar to ethanol, the alcohol in adult beverages. Methanol gets into hand sanitizers when the ethanol ingredient is contaminated with methanol. When ethanol is made by distilleries, sometimes inadvertently methanol is distilled into the product, so then it becomes part of the hand sanitizer.

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How can an oral drug successfully treat the genetic disorder cystic fibrosis?

By

On August 14, 2020

In cellular basis of life, chemical basis of life, digestive system, respiratory system, Uncategorized

TeachableMedicalNews article 08142020

Teachable moment in classrooms:

  1. chemical basis of life chapter – protein structure and shape is important for proper function
  2. cellular basis of life chapter – ion channel proteins in plasma membrane
  3. cellular basis of life chapter – concept of gene mutation leading to protein malfunction
  4. cellular basis of life chapter – involvement of the smooth endoplasmic reticulum apparatus in protein processing
  1. tissue chapter –function of ciliated epithelium
  2. respiratory system chapter – anatomy of the conduction zone
  3. respiratory system chapter – the lining of bronchi and bronchioles

The news item:  Recently a new treatment was reported for cystic fibrosis:

Studies Yield ‘Impressive’ Results in Fight Against Cystic Fibrosis (Published 2019)

The findings hold promise for a vast majority of those with cystic fibrosis, according to the director of the National Institutes of Health. “This should be a cause for major celebration,” he wrote.

The article mentions that cystic fibrosis is a genetic disorder, and it affects the lungs and bowels of the patients.

So, Why Do I Care??  Over 30,000 people in the USA has cystic fibrosis, and 1000 new cases are diagnosed each year.  The mean life expectancy is age 44 for cystic fibrosis patients.  Only symptomatic treatments have been available until now, because gene therapy has been unsuccessful. Any treatment that reverses the root cause of this disorder is a revolutionary achievement that will literally breathe new life into patients.

Plain English, Please!!! First let’s talk about cystic fibrosis. This disorder causes

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Can a self-injected treatment help asthma sufferers?

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On July 26, 2020

In cardiovascular system, cellular basis of life, chemical basis of life, respiratory system, tissues

TeachableMedicalNews article 07262020

Teachable moment in classrooms:

  1. chemical basis of life chapter – protein structure determines biological activity
  2. cellular basis of life chapter – concept of receptor proteins in cell membranes
  3. tissue chapter – locations of smooth muscle
  4. immune system chapter – cytokines as stimulators of immune functions
  5. cardiovascular system chapter – white blood cell types in circulating blood
  6. respiratory system chapter – dilation and constriction of bronchioles

The news item:  Recently a new form of asthma treatment was in the news:

‘Life-changing’ asthma injection will be offered to patients

Around 100,000 patients in the UK whose symptoms are too bad for traditional inhalers or steroids will be eligible for treatment with benralizumab.

The article mentions the disorder eosinophilic asthma, that it is caused by too many white blood cells in the lungs, and that this asthma limits what sufferers may do in their day to day lives.

So, Why Do I Care??  Asthma is a respiratory disorder that afflicts 2.5 million people in the USA. In the age group of 35-50 year olds the eosinophilic asthma is the most frequent. Inhaled pharmaceuticals, common for asthma treatment, come with undesirable side effects after long-term use.  Attacking asthma from a different angle provide a new way to treat patients, and it may help people where inhaled asthma treatments fail.

Plain English, Please!!! First, let’s talk about asthma. While asthma may be started by different conditions, like allergies, cold temperature, the root cause of asthma is always the unwanted restriction of airflow into the lungs.  An important part of our respiratory system is made up of tubes that channel air deep into our lungs, to the air sacks. A set of those tubes, the bronchioles, are 1/20 of an inch in diameter, and they have the ability to narrow and restrict airflow, because the bronchioles have smooth muscle in their walls. When those smooth muscles contract, the narrowed bronchiole restricts airflow to the air sacks.

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Could a targeted blockage of cell division become a new way to treat breast cancer ?

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On July 16, 2020

In cellular basis of life, chemical basis of life, reproductive system

TeachableMedicalNews article 07162020

Teachable moment in classrooms:

  1. chemical basis of life – tertiary structure of proteins
  2. cellular basis of life chapter – cell cycle
  3. cellular basis of life chapter – stages of mitosis
  4. reproductive system chapter – structures of the mammary gland

The news item:  Recently a new form of cancer treatment was in the news:

Newer drug extends lives of young breast cancer patients, study finds

The drug Kisqali (ribociclib) is already approved by the FDA, and new research shows it helps some breast cancer patients live longer

The article states that the drug Kisquali extended the survival time of breast cancer patients, and that the drug is a CDK4/6 inhibitor.

So, Why Do I Care??  In the USA alone there will be over 270,000 breast cancer cases in 2020, and it is expected that more than 47,000 breast cancer deaths will happen.  Any improvement in the survival rate over the treatment by traditional chemotherapy is good news.  In addition, attacking breast cancer from a new direction may improve the treatment of other cancers as well.

Plain English, Please!!! First, let’s talk about breast cancer. This cancer is not actually the cancer of the entire breast. Within the breast tissue there is a gland called mammary gland that makes milk in mothers after giving birth. The milk is made in deeper segments of the gland, and several tubes called lactiferous ducts bring the milk to the skin surface. It is the cells of those ducts that develop cancer in most breast cancers.

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