Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
Gene silencing is the regulation of gene expression in a cell to prevent the expression of a certain gene. Gene silencing can occur during either transcription or translation and is often used in research. In particular,methods used to silence genes are being increasingly used to produce therapeutics to combat cancer and other diseases,such as infectious diseases and neurodegenerative disorders.
Telomerase,also called terminal transferase,is a ribonucleoprotein that adds a species-dependent telomere repeat sequence to the 3' end of telomeres. A telomere is a region of repetitive sequences at each end of the chromosomes of most eukaryotes. Telomeres protect the end of the chromosome from DNA damage or from fusion with neighbouring chromosomes. The fruit fly Drosophila melanogaster lacks telomerase,but instead uses retrotransposons to maintain telomeres.
Small interfering RNA (siRNA),sometimes known as short interfering RNA or silencing RNA,is a class of double-stranded RNA at first non-coding RNA molecules,typically 20–24 base pairs in length,similar to miRNA,and operating within the RNA interference (RNAi) pathway. It interferes with the expression of specific genes with complementary nucleotide sequences by degrading mRNA after transcription,preventing translation.
Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms,including ribonuclease H mediated decay of the pre-mRNA,direct steric blockage,and exon content modulation through splicing site binding on pre-mRNA. Several ASOs have been approved in the United States,the European Union,and elsewhere.
Cancer immunotherapy (immuno-oncotherapy) is the stimulation of the immune system to treat cancer,improving on the immune system's natural ability to fight the disease. It is an application of the fundamental research of cancer immunology and a growing subspecialty of oncology.
A short hairpin RNA or small hairpin RNA is an artificial RNA molecule with a tight hairpin turn that can be used to silence target gene expression via RNA interference (RNAi). Expression of shRNA in cells is typically accomplished by delivery of plasmids or through viral or bacterial vectors. shRNA is an advantageous mediator of RNAi in that it has a relatively low rate of degradation and turnover. However,it requires use of an expression vector,which has the potential to cause side effects in medicinal applications.
Toll-like receptor 8 is a protein that in humans is encoded by the TLR8 gene. TLR8 has also been designated as CD288. It is a member of the toll-like receptor (TLR) family.
Adoptive cell transfer (ACT) is the transfer of cells into a patient. The cells may have originated from the patient or from another individual. The cells are most commonly derived from the immune system with the goal of improving immune functionality and characteristics. In autologous cancer immunotherapy,T cells are extracted from the patient,genetically modified and cultured in vitro and returned to the same patient. Comparatively,allogeneic therapies involve cells isolated and expanded from a donor separate from the patient receiving the cells.
Ram I. Mahato is a professor and chairman of the Department of Pharmaceutical Sciences,University of Nebraska Medical Center,Omaha,United States. He was Professor of Pharmaceutical Sciences and Biomedical Engineering at the University of Tennessee Health Science Center,Memphis. He was Research Assistant Professor at the University of Utah,Senior Scientist at GeneMedicine Inc and a postdoctoral fellow at the University of Southern California,Washington University in St. Louis,and Kyoto University. He received a PhD in drug delivery from the University of Strathclyde and BS from China Pharmaceutical University. He is a CRS Fellow,AAPS Fellow,Permanent Member of BTSS/NIH Study section (2009–2013),and ASGCT Scientific Advisor.
Hua Eleanor Yu is the inaugural Billy and Audrey L. Wilder Professor in tumor immunotherapy at the Beckman Research Institute of the City of Hope National Medical Center in Duarte,California. In addition,she co-leads the Cancer Immunotherapeutics Program at the City of Hope cancer center,with Peter P. Lee. Yu's laboratory was the first to identify STAT3,a protein that helps to protect tumor cells from the immune system. Her group is developing possible drug treatments using CpG-Stat3 siRNA to attack tumor cells in mice and humans.
Weiping Zou is the Charles B. de Nancrede Professor of Pathology,Immunology,Biology,and Surgery at the University of Michigan. He is a scientist noted for his work regarding understanding the nature of human tumor immune responses and developing mechanism-informed combination therapies for cancer. He has developed an international reputation in human tumor immunosuppressive mechanisms in the tumor microenvironment.
Julianna Lisziewicz is a Hungarian immunologist. Lisziewicz headed many research teams that have discovered and produced immunotheraputic drugs to treat diseases like cancer and chronic infections like HIV/AIDS. Some of these drugs have been successfully used in clinical trials.
The mutanome is the entirety of somatic cancer mutations in an individual tumor.
Antibody-oligonucleotide conjugates or AOCs belong to a class of chimeric molecules combining in their structure two important families of biomolecules:monoclonal antibodies and oligonucleotides.
Duane A. Mitchell is an American physician-scientist and university professor. He is currently employed at the University of Florida College of Medicine,in Gainesville,Florida as the Assistant Vice President for Research,Associate Dean for Translational Science and Clinical Research,and Director of the University of Florida (UF) Clinical and Translational Science Institute. He is the Phyllis Kottler Friedman Professor in the Lillian S. Wells Department of Neurosurgery. and co-director of the Preston A. Wells Jr. Center for Brain Tumor Therapy. Mitchell is also the founder,President,and Chairman of iOncologi,Inc.,a biotechnology company in Gainesville,FL specializing in immuno-oncology.
Michel Sadelain is an genetic engineer and cell therapist at Memorial Sloan Kettering Cancer Center,New York,New York,where he holds the Steve and Barbara Friedman Chair. He is the founding director of the Center for Cell Engineering and the head of the Gene Transfer and Gene Expression Laboratory. He is a member of the department of medicine at Memorial Hospital and of the immunology program at the Sloan Kettering Institute. He is best known for his major contributions to T cell engineering and chimeric antigen receptor (CAR) therapy,an immunotherapy based on the genetic engineering of a patient's own T cells to treat cancer.
RNA therapeutics are a new class of medications based on ribonucleic acid (RNA). Research has been working on clinical use since the 1990s,with significant success in cancer therapy in the early 2010s. In 2020 and 2021,mRNA vaccines have been developed globally for use in combating the coronavirus disease. The Pfizer–BioNTech COVID-19 vaccine was the first mRNA vaccine approved by a medicines regulator,followed by the Moderna COVID-19 vaccine,and others.
Uğur Şahin is a German oncologist and immunologist. He is the founder and CEO of BioNTech,which developed one of the major vaccines against COVID-19. His main fields of research are cancer research and immunology.
Cellular adoptive immunotherapy is a type of immunotherapy. Immune cells such as T-cells are usually isolated from patients for expansion or engineering purposes and reinfused back into patients to fight diseases using their own immune system. A major application of cellular adoptive therapy is cancer treatment,as the immune system plays a vital role in the development and growth of cancer. The primary types of cellular adoptive immunotherapies are T cell therapies. Other therapies include CAR-T therapy,CAR-NK therapy,macrophage-based immunotherapy and dendritic cell therapy.
