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The technology of gene therapy is possible due to extensive DNA research and our resulting understanding of many diseases on the genetic level, and it encompasses several mechanisms such as introducing new genes (gene addition) and inactivating or replacing mutated genes (gene editing). Formulation Considerations.
Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with geneticdiseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency.
Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat geneticdiseases. Researchers will compare the two-time points to recognize the optimum timing of peak immuneresponse following nasal vaccination.
Verily collaborates with Janssen to launch COVID-19 immuneresponse study. Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immuneresponses to a coronavirus infection, with people participating in the research from within their homes. .
AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited geneticdisease that causes cardiac and central nervous system dysfunction.
Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males. Patients with this disease have a very high level of LDL-C, which increases their risk of coronary artery disease, including heart attack, stroke and atherosclerosis.
GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative geneticdiseases that includes Tay-Sachs and Sandhoff diseases. aTyr Pharma completed target enrollment in its Phase Ib/IIa trial of ATYR1923 in pulmonary sarcoidosis, a form of interstitial lung disease.
In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. These could feature in the next four to five years.
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