The Theme of Our Second Issue: Advances in Gene and Gell Therapy
The Rise of Gene and Cell-Based Therapies
Over the last couple of decades, the scientific community has made ground-breaking advancements, perhaps some of the most notable being gene and cell-based therapies. As a result of the many technological developments, the prospect for more innovative therapies has been greatly augmented. Gene therapy involves altering genetic components such as DNA and RNA as a therapeutic strategy to treat and prevent diseases. Cell-based therapy ranges from more common procedures such as blood transfusions to those much more complex such as CAR-T cell therapy. The basis of these therapies is infusing new or modified cells into a patient to modulate a particular disease state. Given the unlimited number and combination of mutations that can take place in the human body, innovative approaches such as these will undoubtedly be at the crux of how diseases are treated in the years to come.
FDA-Approved Gene Therapies
As of June, last year, there are currently two gene therapies that have been FDA-approved. Both therapies involve the utilization of an adeno-associated virus vector, one with a focus on inherited retinal dystrophy and the other targeting spinal muscular atrophy. In terms of cell therapies, CAR-T cell therapies have been transformative, particularly in the treatment of cancer.
Inherited retinal diseases (IRDs) have limited therapeutic options as of now. With the momentum that gene therapy is gaining, the future of the treatment of these diseases is reassuring. Notably, FDA-approved for RPE-65 mutation-associated retinal dystrophy, is LUXTURNA. This retinal-pigment encoding gene is directly involved in the process of phototransduction therefore any mutations manifest as visual impairment.
Spinal muscular atrophy is a neuromuscular disease resulting from a mutation in the SMN1 gene. The condition is characterized by severe muscle weakness which can lead to additional complications such as respiratory difficulty, obesity, and an increased risk for infections. In 2019, the FDA approved the gene therapy Zolgensma which replaces the defective copy of the SMN1 gene.
CAR-T Cell Therapy
CAR-T cell therapy has become increasingly popular in the management of cancers that are resistant to the traditional treatment modalities such as chemotherapy and monoclonal antibodies. This type of therapy relies on T cells which are responsible for carrying out various immune responses in the body. Blood is drawn from patients to separate these cells and engineer them such that they express chimeric antigen receptors (CARs). The CARs facilitate T cell recognition of tumor cell antigens.
Publish with Us
As you can see from these few examples, the transformative nature of gene and cell therapies is the reason why Berkeley Pharma Tech has chosen this to be the focus for the second edition of the BPT Journal of Medicine. Being an online, free-to-publish, and open-access journal, this is a unique opportunity for researchers to showcase their work and contribute to the growing wealth of knowledge within the scientific community. We are currently looking for novel research as well as reviews of the latest scientific developments, ideally in the areas of gene and cell therapies. Participants can also submit public policy manuscripts, book reviews, or cartoons which relate to scientific content. The submission deadline is April 15th and issue release date is June 17, 2022.
If you have any questions, please do not hesitate to contact our Editor-in-Chief, Kumari Nallakumar, Ph.D. at kumari@berkeleypharmatech.com.
