Gaucher+4

= Prevention of Gaucher Disease  = Can gene therapy be used??? Since Gaucher Disease is caused by the absence of specific enzymes, Patients with this condition are missing or have defective genes needed for the normal production of these enzymes. Studies on the blood-forming cells in bone marrow have lead to gene therapies using retroviruses as vehicles to carry and insert working genes into abnormal or diseased cells. The figure below illustrates that gene therapy is the practice of injecting DNA directly into cells to correct a genetic defect. The treatment of disease by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is responsible for a disease (7).

In 2009, studies were performed on lab mice  to research the use of gene therapy on Type II Gaucher Disease. The mice were injected with common mutations found in GD patients into their genomes by "knockin" strategies. Unfortunately, majority of the resulting mice died immediately after birth, or had minimal accumulation of GluCer and no clinical indication of GD. Gene therapy is ongoing science that is always being experimented with to help cure the most devastating of disease.

Treatment
 * Enzyme replacement therapy is now available to help stop progression, and often reversing symptom of patients. Every two weeks, individuals go in for an hour session of glucocerbrosidase intravenously being injected into their systems (3).

Other clinical treatments include:
 * Removal of spleen
 * Blood transfusions
 * Pain medications
 * Joint Replacement Surgery

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