Unraveling the Cure

PRESIDENT'S NOTE produce biologic traits, like eye color, height, or even susceptibility to cancer. Studies around the world showed that deoxyribonucleic acid (DNA) was the chemical foundation of chromosomes and the location of genetic information. By 1952, Rosalind Franklin and Raymond Gosling at Cambridge University, had described the three dimensional structure of DNA and in 1953, James Watson and Francis Crick at Cambridge described its chemical structure as a double helix, for which they won the Nobel Prize in Physiology and Medicine. Unraveling Once it was understood that DNA codes for our traits, efforts to decipher that code began. Studies found that four unique molecules—Adenosine, Guanine, the Cure Thymidine and Cytosine—were the building blocks of DNA. Each of these molecules is Solomon I. Hamburg, A brief history of molecular genetics linked together in a specific order of three MD-PhD to determine the information that results in specific biologic traits. In the 1980’s, Paul Berg, Walter n 1865, an Augustine monk named University, studied the Fruit Fly and Gilbert and Frederick Sanger developed Gregor Mendel described the found that genes, or packets of transmittal methods of mapping the structure of characteristics of peas and pea pods as information, could be found on the DNA. Meanwhile, Dr. Stanley Norman being derived from the characteristics of chromosomes located in the nucleus Cohen and Dr. Herbert Boyer received the Itheir parents. Yellow pea plants, cross-bred of cells. first U.S. patent for gene cloning. They with other yellow pea plants led to yellow successfully placed a human gene into pea plants—this much was obvious. But Having recognized that biologic traits are bacteria and produced a "protein foreign yellow plants cross-bred with green plants passed down from generation to generation, to a unicellular organism." These two yielded ¾ yellow peas and ¼ green peas. the study of population genetics was born. scientists were able to replicate proteins The results of these experiments led to the Using mathematical models and statistics, such as HGH, Erythropoietin and Insulin, science of genetics and the understanding the frequency of genetic traits within producing them in bacterial cultures. of how information from one parent of any populations could be ascertained and even The patent earned about $300 million in organism is transmitted to its offspring in a predicted. licensing royalties for Stanford University. predictable manner. In 1941, Edward Lawrie Tatum and In 1987, Yoshizumi Ishino accidentally Between 1915 and 1925, Dr. Thomas Hunt George Wells Beadle showed that genes discovered and described part of a Morgan, a researcher at Johns Hopkins code for proteins. It is these proteins that DNA sequence which would later be called CRISPR, and by 2001 Francisco 6 | TOWER | SPRING 2018 PRESIDENT'S NOTE Mojica and Rudd Jansen used this DNA THE HISTORY EGFR mutation, Zykadia for patients with sequence to successfully edit genes the ALK mutation, and Levantinib for within organisms. OF GENETICS patients with TKI mutations. 1 In 1865, Gregor Mendel, Later, in 1989 Thomas Cech showed through his work on pea plants, The promising CRISPR technology is being that RNA carries the information stored discovered the fundamental laws of evaluated to splice out genetic mutations in DNA, thereby completing the “Central inheritance. and correct them. RNA inhibitors are being Dogma” of molecular biology: DNA makes studied as therapy for leukemia and other 2 Between 1915 and 1925, RNA makes protein. It is considered one Dr. Thomas Hunt Morgan's malignancies. of the most experimental research with the fruit important fly (Drosophila) established the So where are we headed? I can tell you that breakthroughs chromosome theory of heredity. molecular biology and molecular genetics are in molecular 3 In 1941 Edward Lawrie Tatum and the future of medical care. Studies looking genetics. George Wells Beadle showed that for molecular changes within a cancer cell genes code for proteins, the origin are readily In the 125 of the central dogma of genetics. "So where are available to years from all patients. 4 In 1952, Rosalind Franklin and we going? I Mendel to Raymond Gosling decribed the Ways in Cech, our three dimensional structure of which we DNA. can tell you understanding that molecular can use that of the 5 In 1953, James Watson and information transmission of genetic traits of all biologic Francis Crick decribed the double biology and for organisms has built a foundation for helix, for which they won the Nobel prognosis, Prize in Physiology and Medicine. attacking some of our greatest ailments. molecular treatment 6 In the 1980’s Paul Berg, Walter genetics are and In recent decades, great strides have been Gilbert and Frederick Sanger counseling made in the study of cancer biology. developed method of mapping the the future of are being structure of DNA. Recognition that changes in the genetic medical care." aggressively code, or mutations, can lead to loss or gain 7 In 1987, Yoshizumi Ishino studied. of protein function, which in turn lead accidentally discovered part of to cancer, has led to important medical a DNA sequence which later This year’s campaign to raise $5 million for would be called CRISPR and by advances in cancer care. Laboratory testing 2001 Franciso Mojica and Rudd research in molecular genetics is important is now available to screen for these mutated Jansen used this DNA sequence in moving these studies forward. Each genes. For example, in the well-known to successfully edit genes within discovery leads to another, and it always BRACA1 and BRACA2 genes, mutations organisms. starts in the laboratory of a scientist that are associated with an increased risk of 8 In 1989, Thomas Cech shows wants to ask a basic question, like what breast, ovarian and colon cancer. These that RNA can catalyze chemical determines a pea’s color. mutations can be easily evaluated and reactions and thereby completing the central dogma of molecular spotted early on. biology. I believe that we are heading in a direction where our understanding of the molecular Many molecular targets have been developed changes that lead to disease will eventually and have come to market, including lead to a cure for all cancers. Herceptin for patients with the Her 2 neu mutation, Tarceva for patients with the Please help us move forward. SPRING 2018 | TOWER | 7.

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