The excitement surrounding cell and gene therapies predominantly stems from their potential to treat and cure diseases previously considered untreatable. This includes many rare genetic diseases such as Duchenne muscular dystrophy or hemophilia A, as well as various types of cancer. Take for instance the story of Caroline and Cole Carper, who were born with …

Advances in gene therapies have provided opportunities to potentially correct the genetic abnormalities that lead to rare diseases, thereby offering a cure.

Cell and gene therapies (CGT) have changed the landscape of precision medicine and continue to emerge at a rapid pace. These therapeutic strategies are both specific and precise compared to prior conventional medicines, and offer tremendous potential for the treatment of syndromes and diseases caused by genetic mutations. The personalized nature of CGT approaches requires …

Cell and gene therapies are promising treatment strategies for many diseases. A multifaceted in vitro and in vivo approach is applicable in treatments ranging from cancer to genetic diseases. These therapies rely on transgenes being delivered into specific cell types, therefore identification of cell types and effective use of delivery vehicles is critical. The active …

PerkinElmer recently discussed next-generation cancer therapies with one of the founders of the field, Dr. Sunil Chada. The complexity of cancer continues to pose significant challenges for researchers in the development of novel therapeutic agents. Historic treatment approaches including chemotherapy, radiation, and immunotherapies such as antibody therapies have failed to deliver curative outcomes for most …

Modifying the expression of genes for therapeutic purposes – a process known as gene therapy – has gained attention in recent years due to its promise for treating various genetic disorders. The technique involves replacement, inactivation, or introduction of new genes by a vector that will deliver the gene into the cells and incorporate those …