Human Genetic Diseases

Francisco Said:

Why has gene therapy been unsuccessful in curing human genetic diseases in the past?

We Answered:

Gene therapy involves the insertion of genes or genetic information into a patient's cells in an atempt to treat diseases, usually those related to heritable genetic aberrations/mutations. Some major issues confronting the successful insertion of such genes are:

-the fact that, due to how rapidly many cells in humans divide, current gene therapies can only be inserted into a small number of the host's cells.
-the fact that the host's immune system attacks the viruses used to insert therapeutic genes.
-the problems viral vectors present in relation to the patient (host) related to immune response, viral toxicity, allergy, unique reactions to the virus in individual patients.
-many heritable diseases are multi-gene disorders which are far harder to target effectively with modern gene-therapy methods.
-sometimes/often viral vectors hold the potential to induce, as a side effect of their mass, unrestricted implementation (in the body), tumors.

Caroline Said:

Is there evidence to suggest that Human Genetic Engineering would not work in eliminating genetic diseases?

We Answered:

No. Human genetic engineering has already eliminated many genetic diseases. This happened before people even understood genetics. It works through the process of sexual selection, a type of natural selection. People with visually obvious genetic defects have a lower probability of mating. This then reduces the relative abundance of the genes that cause these problems as long as the problems are visible.

Modern genetic screening techniques can help a great deal to eliminate bad genes. It has the capacity to determine if parents are carriers of bad genes and if a fetus has received a set of bad genes. This works only with identified genetic defects. Whether or not direct manipulation of genes will be of great help is still an open question.

Russell Said:

bio final tomorro!!!!!! 10 pts plz help!!! racial and ethnic prevelance over certain human genetic diseases?

We Answered:

Racially, humans as a whole are all equally at risk for genetic diseases. However, when humans live in small isolated populations, inbreeding can lead to the accumulation of random mutations, resulting in certain genetic diseases characteristic of a particular population.

Jews are prone to several genetic disorders, the most famaliar is Tay Sachs syndrome. It is a neurological disorder which kills children before they reach three years of age. During most of the last 2000 years, European Jews have lived isolated lives to their own separate communities, mostly due to religious prejustices.

Sicle cell anemia is common in some African communities. It is postulated this is a semi beneficial mutation in responce to malaria. The deformed shape of red blood cells prevents malaria paracites from infecting the carriers of this disease. However, the deformed blood cells frequently block capillaries, causing pain, poor circulation and even fatal gangrene. However, malaria itself is more lethal in general. In fact malaria kills more people on earth than any other disease.

Hemophilia is another blood disorder which became established in European royal families. This group of people reproduced only within the confines of half a dozen founding individuals, so the mutation became established. Queen Victoria was a carrier of this defect, but to express itself, the defect required the gene to be present on the X and Y sex chromosomes. Hence, only males showed symptoms of the disease because females only have a pair of X chromosomes. Nicolas , the last Romanoff Tzar of Russia had a son which showed signs of this disease. Hemophilia results from a defect in a blood clotting protien. The men who suffer this disease are highly prone to uncontrolled bleeding.

Kimberly Said:

what human diseases are characterized by a genetic component?

We Answered:

Huntington's disease
phenylketoneuria
color blindness
Down syndrome

Terrance Said:

How many types of mutation in human genetic diseases?

We Answered:

Here is a website that will tell u all of them.

http://en.wikipedia.org/wiki/List_of_genetic_disorders

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