Richard C. Mulligan | |
---|---|
Alma mater | Massachusetts Institute of Technology, Stanford University |
Known for | Gene Therapy |
Scientific career | |
Fields | Gene Therapy |
Institutions | Massachusetts Institute of Technology, Harvard Medical School, Sana Biotechnology |
Doctoral advisor | Paul Berg |
Other academic advisors | Alexander Rich, David Baltimore, Phillip Allen Sharp |
Notable students | James Wilson (scientist), Constance Cepko, David Sanders (biologist), Roger D. Cone |
Richard C. Mulligan (born 1954) is an American scientist who is the Mallinckrodt Professor of Genetics at Harvard Medical School, [1] [2] [3] the Director of the Harvard Gene Therapy Initiative and a visiting scientist at the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology. [4] He is also the head of SanaX at Sana Biotechnology.
Mulligan started his career in gene therapy as an undergraduate in biology in Alexander Rich's lab at MIT and was involved with early work controlling gene expression using SV40. He would earn his PhD in biochemistry at Stanford University in 1980 working with Paul Berg to develop viral vectors to express human and bacterial genes. [5] [6] He would then do his postdoctoral training at the Center for Cancer Research at MIT with David Baltimore and Phillip Sharp. He would join the faculty of molecular biology and was a member of the Whitehead Institute for Biomedical Research. [7] During that time, he was a founding member of the Recombinant DNA Advisory Committee (RAC). In 1996, he joined Children's Hospital and Harvard to become the director of the Harvard Gene Therapy Initiative and an investigator of the Howard Hughes Medical Institute. [8]
Mulligan is an active investor as the founding partner and senior managing director of Sarissa Capital Management from 2013 to 2016 along with Alex Denner when they worked together with Carl Icahn. [9] He would then join Icahn Capital as a portfolio manager in 2017. He serves as Director of Enzon Pharmaceuticals, Inc., [10] and Biogen Idec, Inc. [11] [12]
Genetic engineering, also called genetic modification or genetic manipulation, is the modification and manipulation of an organism's genes using technology. It is a set of technologies used to change the genetic makeup of cells, including the transfer of genes within and across species boundaries to produce improved or novel organisms. New DNA is obtained by either isolating and copying the genetic material of interest using recombinant DNA methods or by artificially synthesising the DNA. A construct is usually created and used to insert this DNA into the host organism. The first recombinant DNA molecule was made by Paul Berg in 1972 by combining DNA from the monkey virus SV40 with the lambda virus. As well as inserting genes, the process can be used to remove, or "knock out", genes. The new DNA can be inserted randomly, or targeted to a specific part of the genome.
Molecular genetics is a sub-field of biology that addresses how differences in the structures or expression of DNA molecules manifests as variation among organisms. Molecular genetics often applies an "investigative approach" to determine the structure and/or function of genes in an organism's genome using genetic screens. The field of study is based on the merging of several sub-fields in biology: classical Mendelian inheritance, cellular biology, molecular biology, biochemistry, and biotechnology. Researchers search for mutations in a gene or induce mutations in a gene to link a gene sequence to a specific phenotype. Molecular genetics is a powerful methodology for linking mutations to genetic conditions that may aid the search for treatments/cures for various genetics diseases.
Chinese hamster ovary (CHO) cells are an epithelial cell line derived from the ovary of the Chinese hamster, often used in biological and medical research and commercially in the production of recombinant therapeutic proteins. They have found wide use in studies of genetics, toxicity screening, nutrition and gene expression, particularly to express recombinant proteins. CHO cells are the most commonly used mammalian hosts for industrial production of recombinant protein therapeutics.
William French Anderson is an American physician, geneticist and molecular biologist. He is known as the "father of gene therapy". He graduated from Harvard College in 1958, Trinity College, Cambridge University (England) in 1960, and from Harvard Medical School in 1963. In 1990 he was the first person to succeed in carrying out gene therapy by treating a 4-year-old girl suffering from severe combined immunodeficiency. In 2006, he was convicted of sexual abuse of a minor and in 2007 was sentenced to 14 years in prison. He was paroled on May 17, 2018, for good behavior.
Recombinant DNA (rDNA) molecules are DNA molecules formed by laboratory methods of genetic recombination that bring together genetic material from multiple sources, creating sequences that would not otherwise be found in the genome.
Stanley Norman Cohen is an American geneticist and the Kwoh-Ting Li Professor in the Stanford University School of Medicine. Stanley Cohen and Herbert Boyer were the first scientists to transplant genes from one living organism to another, a fundamental discovery for genetical engineering. Thousands of products have been developed on the basis of their work, including human growth hormone and hepatitis B vaccine. According to immunologist Hugh McDevitt, "Cohen's DNA cloning technology has helped biologists in virtually every field". Without it, "the face of biomedicine and biotechnology would look totally different." Boyer cofounded Genentech in 1976 based on their work together, but Cohen was a consultant for Cetus Corporation and declined to join. In 2022, Cohen was found guilty of having committed fraud in misleading investors into a biotechnology company he founded in 2016, and paid $29 million in damages.
Tom Maniatis, is an American professor of molecular and cellular biology. He is a professor at Columbia University, and serves as the Scientific Director and CEO of the New York Genome Center.
Philip Leder was an American geneticist.
Viral vectors are tools commonly used by molecular biologists to deliver genetic material into cells. This process can be performed inside a living organism or in cell culture. Viruses have evolved specialized molecular mechanisms to efficiently transport their genomes inside the cells they infect. Delivery of genes or other genetic material by a vector is termed transduction and the infected cells are described as transduced. Molecular biologists first harnessed this machinery in the 1970s. Paul Berg used a modified SV40 virus containing DNA from the bacteriophage λ to infect monkey kidney cells maintained in culture.
Krueppel-like factor 1 is a protein that in humans is encoded by the KLF1 gene. The gene for KLF1 is on the human chromosome 19 and on mouse chromosome 8. Krueppel-like factor 1 is a transcription factor that is necessary for the proper maturation of erythroid cells.
A genetically modified virus is a virus that has been altered or generated using biotechnology methods, and remains capable of infection. Genetic modification involves the directed insertion, deletion, artificial synthesis or change of nucleotide bases in viral genomes. Genetically modified viruses are mostly generated by the insertion of foreign genes intro viral genomes for the purposes of biomedical, agricultural, bio-control, or technological objectives. The terms genetically modified virus and genetically engineered virus are used synonymously.
Robert J. Desnick is an American human geneticist whose basic and translational research accomplishments include significant discoveries in genomics, pharmacogenetics, gene therapy, personalized medicine, and the treatment of genetic diseases. His translational research has led to the development the enzyme replacement therapy (ERT) and the chaperone therapy for Fabry disease, ERT for Niemann–Pick disease type B, and the RNA Interference Therapy for the Acute Hepatic Porphyrias.
Molecular cloning is a set of experimental methods in molecular biology that are used to assemble recombinant DNA molecules and to direct their replication within host organisms. The use of the word cloning refers to the fact that the method involves the replication of one molecule to produce a population of cells with identical DNA molecules. Molecular cloning generally uses DNA sequences from two different organisms: the species that is the source of the DNA to be cloned, and the species that will serve as the living host for replication of the recombinant DNA. Molecular cloning methods are central to many contemporary areas of modern biology and medicine.
Richard M. Myers is an American geneticist and biochemist known for his work on the Human Genome Project (HGP). The National Human Genome Research Institute says the HGP “[gave] the world a resource of detailed information about the structure, organization and function of the complete set of human genes.” Myers' genome center, in collaboration with the Joint Genome Institute, contributed more than 10 percent of the data in the project.
Genetic engineering is the science of manipulating genetic material of an organism. The first artificial genetic modification accomplished using biotechnology was transgenesis, the process of transferring genes from one organism to another, first accomplished by Herbert Boyer and Stanley Cohen in 1973. It was the result of a series of advancements in techniques that allowed the direct modification of the genome. Important advances included the discovery of restriction enzymes and DNA ligases, the ability to design plasmids and technologies like polymerase chain reaction and sequencing. Transformation of the DNA into a host organism was accomplished with the invention of biolistics, Agrobacterium-mediated recombination and microinjection. The first genetically modified animal was a mouse created in 1974 by Rudolf Jaenisch. In 1976 the technology was commercialised, with the advent of genetically modified bacteria that produced somatostatin, followed by insulin in 1978. In 1983 an antibiotic resistant gene was inserted into tobacco, leading to the first genetically engineered plant. Advances followed that allowed scientists to manipulate and add genes to a variety of different organisms and induce a range of different effects. Plants were first commercialized with virus resistant tobacco released in China in 1992. The first genetically modified food was the Flavr Savr tomato marketed in 1994. By 2010, 29 countries had planted commercialized biotech crops. In 2000 a paper published in Science introduced golden rice, the first food developed with increased nutrient value.
Eric Emil Schadt is an American mathematician and computational biologist. He is founder and former chief executive officer of Sema4, a patient-centered health intelligence company, and dean for precision medicine and Mount Sinai Professor in Predictive Health and Computational Biology at the Icahn School of Medicine at Mount Sinai. He was previously founding director of the Icahn Institute for Genomics and Multiscale Biology and chair of the Department of Genetics and Genomics Sciences at the Icahn School of Medicine at Mount Sinai.
Margaret (“Peggy”) A. Goodell is an American scientist working in the field of stem cell research. Goodell is Chair of the Department of Molecular and Cellular Biology at Baylor College of Medicine, Director of the Stem Cell and Regenerative Medicine (STaR) Center, and a member of the National Academy of Medicine. She is best known for her discovery of a novel method to isolate adult stem cells.
Clifford James Tabin is chairman of the Department of Genetics at Harvard Medical School.
Keiichi Itakura is an organic chemist and a Professor in the Department of Molecular and Cellular Biology at the Beckman Research Institute at City of Hope National Medical Center.
Edward F. Fritsch is a scientist in the field of molecular biology and cancer immunology.