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Scientific Breakthroughs - Gene Therapy for Genetic Diseases and Disorders - Hope, Help, and Headway

Updated on April 7, 2011

Genes Make Us Unique

We are all  special. Our genes are part of our uniqueness. But when it comes to a disease, we don’t want to be unique. Many diseases are the result of genes that are flawed. Gene therapy replaces bad genes with good genes. It is this new therapy that gives promis and hope for cures and relief of ailments that alter the lives of people who are afflicted with a disorder.

Parkinson's Disease is a Genetic Disorder

Parkinson’s disease (PD)is a progressive disorder of the central nervous system. It affects movement, is degenerative, and is chronic. It affects 1% of people over 60, although some have been diagnoses as young as 18 years old. People who suffer from Parkinson’s disease lose brain cells that create the chemical dopamine. Dopamine transmits signals in the brain that relate to coordination and movement. Loss of this chemical makes the neurons to misfire and the person loses the ability to control their own movements.
Currently the way Parkinson’s disease is treated, drugs are used that act like dopamine. However, because neurons are constantly being lost, the drugs eventually lose their effectiveness.

There are about 60,000 Americans who are diagnosed each year, with thousands more going undetected. There are no tests that can confirm or deny that a person has Parkinson’s. It is generally estimated that over 6 million people worldwide have Parkinson’s, including 1 million in the U.S. Parkinson’s disease is believed to be caused by genes that are malfunctioning. It one of many biological genetic disorders.

What are Genes?

Genes tell each cell what specific proteins to make.  These proteins affect every function of our body, from making enzymes that help us digest food to our physical traits of eye and hair color.  Every person has 25,000 to 35,000 different genes that make up their body.

What is Gene Therapy?

There are many medical issues that result from mutations or flaws in the genes that makeup our body. Changes in the structure of  genes can cause a mutation which will make other things in the body go awry. The mutated gene can also be passed down to future generations. Gene mutations are called genetic disorders.

When a person has a disease or disorder, medical science usuall attempts to correct it with drugs. As we make new advances in technology, gene therapy is becoming another alternative. Gene therapy attempts to replace the mutated gene with a functional, normal copy of the gene. It it works, the genetic flaw is corrected.  However, gene therapy is complex and and there is still much to be learned. Scientists have not identified every single gene in the body, and do not know what each gene does and how it functions.

So when they attempt gene therapy, they need to know how that particular gene may affect other parts of the body.

There are Many Genetic Disorders

A new gene therapy being used on people at the Weill Cornell Medical College, who have Parkinson’s has been showing great promise . The particular experiment showed a in significant  easing of the involuntary motor functions that typify this disorder.

Forty five  people with mild to advanced PD were part of an experimental study. 22 were given the new gene therapy, 23 were part of a placebo therapy treatment.  No one, not even the evaluators, were told who was part of which group. Six months later, those who were part of the gene therapy group, remarkably demonstrated a 23.1% improvement in motor scores. Motor scores are an evaluation of their tremors, balance, walking difficulties, and muscle spasms. There was a 12.7% improvement in the other group. The researchers are awaiting approval  to test the next phase of the experiment. So far they are very hopeful that these results will lead to possibly reversing the symptoms  of Parkinson’s disease. Scientists do not view this a cure because they don’t know the fundamental causes of the disorder and why the neurons degenerate. But this experiment is making great strides forward in the treatment of Parkinson’s.

As science make inroads and new discoveries many inherited disorders have the possibility of being alleviated. There are thousands of diseases classified as genetic disorders, some of the most common are:

  • cycstic fibrosis
  • hemophilia
  • cancer
  • sickle cell anemia
  • parkinson’s disease
  • huntington’s disease’

How does Gene Therapy Work?

Gene therapy treatments were first tested in human beings in 1990. It has made tremendous progress in the decade since it was first done. Gene therapy can be done in or outside the body. When done inside a person, doctors use a virus that carries the gene and injects it into the part of the body where the defective cells exist. This is used primarily when a select group of cells can be targeted. This is used for Parkinson’s because a region of the brain is specifically aimed at. It is also used for hemophilia and to treat genetic eye diseases.  The virus that is used has no known ill affects and  is used as a carrier of the gene replacement.  Gene therapy is showing positive effectiveness for hemophilia because blood clotting factors can be add to the blood in the liver through the virus.

Out of the body gene replacement therapy uses blood or bone marrow from the patient. A gene is added to the immature cells that have been separated. The “new cells” travel to the bone marrow and mature. As they multiply they replace the defective cells.  Scientists are working on this type of therapy for sickle cell anemia.

With each success, medical science is building more successes. As scientists learn more about our genes they will be able to apply this knowledge and find ways to eliminate many ailments and disorders. There is great hope for longevity, good health, and quality of life as we get older. Amazing discoveries are happening everyday.


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    • conradofontanilla profile image


      5 years ago from Philippines

      Dr. Morton Walker lists PD as a free radical disease in his book "The Miracle Healing Power of Chelation Therapy." Degenerative disease is caused by free radicals. I have several Hubs on free radicals. Nerve cells are considered non renewable. At the start a lot of neuron cells are produced but not all of them are put to use, some are held in reserve. When those in use conk out, the reserves take their place. It is essential to protect those in reserve by limiting the population of free radicals with the use of antioxidants, built-in enzyme antioxidants or supplements. Enzyme antioxidants include superoxide dismutase and glutathione peroxidase that can be made by the body through glutathione synthase that make glutathione from nutrition (glutamate, cysteine, cystine and cofactors zinc, selenium, lipoic acid and vitamin B2). The body makes lipoic acid.

    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi Mac, Thanks for stopping by and reading. The science of genetics is fascinating. The more we find out about what genes can do, the more gene therapy can be done and the closer we get to curing or at least alleviating some of the diseases that afflict mankind. I really appreciate your interaction.

    • mackyi profile image

      I.W. McFarlane 

      7 years ago from Philadelphia

      Great hub my friend. I strongly believe faulty genes are responsible for many of the diseases and illnesses that often impact the life of many. Thus genetic research is of paramount importance if we are going to be successful in finding a cure or the prevention of many of these diseases or illnesses. Keep up the good work!

    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi Kath, Thanks for your kind comments. Science is making great strides in genetic research. Tomorrow offers great promise and hope that medicine will get ahead of many diseases. Glad you found this informative.

    • Chatkath profile image


      7 years ago from California

      Thank you for an easy to understand, very informative hub toknowinfo. All of us have family or friends that can benefit from this research and it is so essential that we continue to move forward.

    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi Tamarajo, I am sorry about your granddaughter. I hope medical science will make progress in finding a solution to this disorder. I am glad tis article is helpful to you, and if I should read anything new about Joubert syndrome, I will let you know. Thanks for stopping by and contributing.

    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi CHS, I am glad you stopped by. Gene therapy holds great promises. We will wait and see what new discoveries this science makes.

    • Tamarajo profile image


      7 years ago

      fascinating read. My granddaughter has a genetic disorder called Joubert syndrome. I have since become very interested in learning about genetics and how the work. Your article was helpful.

      I am also happy to hear that they are finding ways to help these conditions.

    • chspublish profile image


      7 years ago from Ireland

      It's great that we're making advance in treating such horrible disases. It's certainly the way forward. Good research here.

    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi Sharyn, I take great pride in my writing, as it is a reflection of who I am. I am a perfectionist when it comes to making sure my info and research is correct, because I want you to be able to rely on my writings, which you can. I appreciate you noticing my efforts and thank you for your kind words.

    • Sharyn's Slant profile image

      Sharon Smith 

      7 years ago from Northeast Ohio USA


      I am very intrigued by the topics you choose to write about and the large amount of effort in research that goes in to your work. Extremely well done! Thank you,


    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi Tom, Gene therapy has great potential for many diseases and ailments. Always glad to see you. Enjoy your weekend too.

    • toknowinfo profile imageAUTHOR


      7 years ago

      Thanks Amillar, You made my day!

    • kashmir56 profile image

      Thomas Silvia 

      7 years ago from Massachusetts

      Hi toknowinfo, so glad to hear that they are making some progress in finding better treatments to help slow or even control this nasty disease .

      Vote up !!!

      Have a great weekend my friend !

    • amillar profile image


      7 years ago from Scotland, UK

      Your hubs always leave me feeling informed and full of hope.

      Up and useful.

    • toknowinfo profile imageAUTHOR


      7 years ago

      It must be awesome to be on the cutting edge of new discoveries.

    • Cogerson profile image


      7 years ago from Virginia

      Great hub.....I have a very close friend who works in this field...I can always tell how proud he is when his group finds something that could help countless people in the future....voted up

    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi Pam, Gene therapy is really quite amazing. It will be interesting to see what they do with it next.

    • toknowinfo profile imageAUTHOR


      7 years ago

      Hi Simone, Thanks for stopping by. Medical science has learned a lot for sure, but they still have a long way to go and it would be nice for them to go faster.

    • Pamela99 profile image

      Pamela Oglesby 

      7 years ago from United States

      This is such a tough disease that it is good that they are making some progress in treatment. I think gene therapy is going to be the answer to many diseases eventuaally.

    • Simone Smith profile image

      Simone Haruko Smith 

      7 years ago from San Francisco

      I'm so excited about the progress we're making in the field of medicine... I just wish we could progress a bit faster, is all!! Hahaa.


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