Genetic testing can be used, in some cases, to identify the defective gene causing lipidoses.
Gene therapy involves inserting a healthy copy of a gene into cells that have a defective copy. This can potentially correct the genetic mutation causing the disease and restore normal cell function. The goal is to treat or prevent genetic disorders by replacing or supplementing missing or defective genes.
Women who are carriers of the defective gene that causes Fabry's disease can also be identified by a blood test.
a new gene which codes for the protein that can repair defective genes is introduced.
gene therapy
In cases where a defective gene is located on the X chromosome, a female can inherit the gene from either parent. If the mother carries one defective X chromosome, she can pass it on to her daughter. Similarly, if the father has the defective gene on his single X chromosome, he will pass it to all his daughters, as they inherit his X chromosome. Therefore, females can receive the defective gene from both maternal and paternal sources.
mutations in a single large gene were identified as causing A-T. Researchers named the gene ATM for A-T, mutated. Subsequent research revealed that ATM has a significant role in regulating cell division
Yes
a new gene which codes for the protein that can repair defective genes is introduced.
The expression of a gene of interest can be ensured by combining it with a gene recessive to it.
A person with only one copy of a defective gene often does not develop the associated disorder due to the presence of a normal copy of the gene, which can provide sufficient functional protein or activity to maintain normal physiological processes. This phenomenon is known as "haplosufficiency," where one functional allele compensates for the defective one. Additionally, many genetic disorders are recessive, meaning that both copies of the gene must be defective for the disorder to manifest.
Gene therapy is a method that aims to cure inherited diseases by providing the patient with correct copy of the defective gene. There are four potential approaches to gene therapy:1)Addition of normal gene to replace the function of defective gene. This is gene replacement orgene augmentation therapy.2)Replacing the defective gene with the correct gene. This isCorrective gene therapy.3)Establishment of alternative pathways that bypass mutant genes function4)Change in regulation of normal or mutant genesThe first two are the basic approaches in gene therapy
gene therapy, in which defective gene is replaced by correct gene with help of virus which work as vechicle for viruses