netic disease is prevalent in modern day society and can be debilitating for many. As medical research helps us understand what causes these disorders and also are starting to understand how to treat and even prevent genetic disease. Many individuals have undergone genetic testing to determinate if they carry the gene for certain genetic mutations. A high profile example of this is when Angelina Jolie had a double mastectomy after learning she carried the BRCA 1 gene hence decreasing here chance of developing cancer. There are ever increasing treatments for genetic disease which include gene therapy.
Severe combined Immunodeficiency (SCID) or more commonly referred to as ‘Bubble Boy Syndrome’ is a rare genetic disorder. Most cases of SCID …show more content…
Gene therapy is a therapeutic strategy that involves modifying a target cell with the effect being to halt or reverse a disease process. Retrovirus vectors are often used for the treatment of XSCID as they have the ability to integrate permanently into the genome of the target cells. Permanency is needed for long-term correction of the genetic disease. The integrated provirus behaves as a gene transcription unit; the therapeutic cells will be copied into daughter cells during cell replication. The retrovirus is integrated by binding a viral envelope protein to cell surface receptors. Infusion or viral and cellular membranes leads to the internalisation of the viral code. Gene therapy has experienced extensive accumulation in this field with gene transfer of hematopoietic stem cell (HSC) using these vectors (NOGUIEZ-HELLIN, …show more content…
ZFN’s are artificial restriction enzymes which fuse finger DNA-binding and DNA-cleavage domain. They can be engineered to target specific DNA sequences and target unique sequences (Carroll, 2008). Trials using this more developed technology, no leukaemia cases have been witnessed to date in ADA-SCID patients, ADA-SCID does not involve the gamma c gene that may be oncogenic when expressed by a retrovirus.
SCID is the first genetic disorder to have undergone successful gene therapy in 1999 since then immune systems of at least 17 children with ADA-SCID and X-SCID have become functioning.
Many people disagree with germ-line therapy, this technology can be used not just to treat genetic disorder but to pick specific physical traits for a child. Society could become less accepting towards people with a genetic disorder if germ-line therapy becomes widespread. Germline therapy can also be very risky as eliminating one genetic disorder could lead to other mutations being introduced with these mutations being passed on to the next generation and further. Genetic disorders could become disorders of the past much like polio has been screened out using immunisations (yourgenome, 2015). Germ-line gene therapy is a permanent solution to genetic disease but is currently outlawed in
Severe combined Immunodeficiency (SCID) or more commonly referred to as ‘Bubble Boy Syndrome’ is a rare genetic disorder. Most cases of SCID …show more content…
Gene therapy is a therapeutic strategy that involves modifying a target cell with the effect being to halt or reverse a disease process. Retrovirus vectors are often used for the treatment of XSCID as they have the ability to integrate permanently into the genome of the target cells. Permanency is needed for long-term correction of the genetic disease. The integrated provirus behaves as a gene transcription unit; the therapeutic cells will be copied into daughter cells during cell replication. The retrovirus is integrated by binding a viral envelope protein to cell surface receptors. Infusion or viral and cellular membranes leads to the internalisation of the viral code. Gene therapy has experienced extensive accumulation in this field with gene transfer of hematopoietic stem cell (HSC) using these vectors (NOGUIEZ-HELLIN, …show more content…
ZFN’s are artificial restriction enzymes which fuse finger DNA-binding and DNA-cleavage domain. They can be engineered to target specific DNA sequences and target unique sequences (Carroll, 2008). Trials using this more developed technology, no leukaemia cases have been witnessed to date in ADA-SCID patients, ADA-SCID does not involve the gamma c gene that may be oncogenic when expressed by a retrovirus.
SCID is the first genetic disorder to have undergone successful gene therapy in 1999 since then immune systems of at least 17 children with ADA-SCID and X-SCID have become functioning.
Many people disagree with germ-line therapy, this technology can be used not just to treat genetic disorder but to pick specific physical traits for a child. Society could become less accepting towards people with a genetic disorder if germ-line therapy becomes widespread. Germline therapy can also be very risky as eliminating one genetic disorder could lead to other mutations being introduced with these mutations being passed on to the next generation and further. Genetic disorders could become disorders of the past much like polio has been screened out using immunisations (yourgenome, 2015). Germ-line gene therapy is a permanent solution to genetic disease but is currently outlawed in