We have collected some insights on gene therapy research progress, development trends, applications, and challenges, aiming to inspire even more innovations in gene therapies.
Despite this wealth of gene targets, treatment options are still extremely limited, and clear "druggable" pathways are not obvious for many of these mutations. However, recent advances in gene therapies are beginning to circumvent this challenge. Each type of CMT is a monogenic disorder, and the...
More than 900 investigational gene therapies are in various stages of research and development, and some researchers estimate that 40 will be on the market by 20222. Because gene therapies are likely to have complex manufacturing and supply chain processes—which have implications for where and ...
bacteria, fungi), engineered site-specific nucleases used for human genome editing, and ex vivo genetically modified human cells,” Unlike the current European definition, in the United States there is no distinction between recombinant or synthetic nucleic acids; both are considered gene therapies. ...
There is almost never one single gene that inevitably leads to a given result. Any genetic data we share are simply clues for further exploration. It all begins with sex. Humans reproduce sexually, which means one cell’s genetic information has to hook up with another cell’s genetic informa...
Q: Let’s begin with an overview of extracellular vesicles (EVs). What are they, and what do they potentially bring to the table for the advanced therapies field? AK: The way I like to describe EVs to folks who may not be familiar with them is that they are ...
Often resistant to treatment. Sometimes drugs or surgery are used if tremors are severe. Current Research Into MS There has been a lot of progress over the years in managing multiple sclerosis, and research is ongoing into new therapies. There are several new avenues of current research. Scienti...
What is the goal of gene therapy? What type of technology is used in gene therapy? Define gene therapy. What are the pros and cons of genetic testing for a couple who carries the genes for cystic fibrosis? What does the process of gene therapy involve? What are the advantages and limitat...
What is the treatment for Hirschsprung disease? What is a underlying disease process? What is an epidemic disease process? What diseases are caused by retroviruses? How many viral vector gene therapies are there? What disease process is haloperidol used to treat? What is Hirschsprung's disease?