Gene therapies for genetic diseases can now heal previously uncurable diseases; one example is Novartis’s treatment of spinal muscular atrophy with its drug Zolgensma.30 Another example is GenSight Biologics’ use of gene therapy to cure blindness.31 CRISPR Therapeutics ...
Nucleotide databases can be used to study and examine gene expression by using the sequence information stored in the databases. Nucleotide databases are also used to identify potential drug targets and develop new therapies for genetic diseases. Nucleotide databases also help in identifying genetic vari...
Autosomal recessive disorders like cystic fibrosis or sickle cell anemia require both copies of an autosomal gene to be mutated for the disorder to manifest. Studying autosomal genetic disorders provides valuable insights into disease mechanisms and paves the way for targeted therapies and genetic ...
immunesystem. Anti-angiogenic therapies destroy blood vessels supporting tumors, whilemolecular targetingfocuses on specific cancer-causing genes. However, cancer cells can develop multidrug resistance, often through the MDR1gene, which encodes anABC transporterthat expels drugs, rendering treatments ...
One example of host-pathogen coevolution is seen in theevolution ofsickle cell anemiain humans, which is thought to have arisen as a result of resistance tomalaria. The sickle cell gene, which causes abnormalred blood cells, also confers some resistance to malaria infection. As a result,indivi...
of MRSA. Penicillin and methicillin work by weakening the wall of the bacterial cell; when the wall is compromised, the osmotic gradient between a bacterial cell’scytoplasmand itsenvironmentforces the cell to lyse (break open). In MRSA the gene acquired through conjugation encodes a protein ...
New medical treatments, such as gene therapies, have the potential to save lives or transform them completely, to provide hope where there is none and to justify years of scientific research. Karen Weintraub, USA TODAY, 24 Apr. 2023 Used to justify the murder of Black and brown people at...
Parkinson’s diseaseGene therapiesGene transferNeurodegenerative diseases, such as Parkinson's disease (PD), offer unique opportunities for the therapeutic application of viral vector gene transfer. In this chapter, we discuss the current state ofdoi:10.1007/978-1-62703-610-8_17...
major structural elements of cells. They also serve as transporters, moving nutrients and other molecules in and out of cells, and asenzymesandcatalystsfor the vast majority ofchemical reactionsthat take place in living organisms. Proteins also formantibodiesandhormones, and they influencegeneactivity....
It comes along with some up-front risk of mortality and a lot of potential side effects, such as graft-versus-host disease and a higher risk of infection because of immunosuppressive drugs. Gene therapies for sickle cell disease come with hope and challenges | Erin Garcia de Jesús | February...