X-linked disorders: These disorders are caused by a mutated gene on the X chromosome. Some examples of X-linked disorders include hemophilia, Duchenne muscular dystrophy, and Fragile X syndrome. Monogenic diseases can range in severity from mild to severe. Some monogenic diseases are fatal, while...
These are the 3 key players that help the CRISPR-Cas9 tech work:Guide RNA: a piece of RNA (a genetic cousin of DNA) that locates the targeted gene. This is engineered in a lab. CRISPR-associated protein 9 (Cas9): the “scissors” that snip the undesired DNA out DNA: the desired ...
The coefficient of variation is similar to that of the body weight in a normal adult population (CV ~ 16%). In a disease like hemophilia, where one factor (FVIII) limits the amount of thrombin formed, the amount that is formed in spite of this limitation still varies, depending upon the...
making it theoretically possible to reduce disease risk. While still experimental, it has been successfully used to treat childhood leukemia and has led to the development of new therapies for hemophilia and cystic fibrosis.
First and primary is the clinical management of patients, because many VWD patients are followed in specialized hemophilia treatment centers. The health care professionals managing patients are highly trained hematologists who have the necessary expertise to evaluate the disease burden. However, and as ...