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"Designing drugs" sounds trendy, but it accurately describes how some remarkably effective drugs are developed. In this case study we'll follow the steps of drug design, from initial research on the targeted disease, to the drug's use in humans. Gleevec (STI-571) is our example.

When a person dies from cancer, the culprit is usually not the original tumor but rather the cancerous cells that spread throughout the body and replicate in distant organs, a process called metastasis. Researchers have long known that metastasizing cancer cells slip their bonds and avoid immune detection by altering the sugars on their surfaces. They've even come up with a would-be drug to prevent such sugar alterations. But that compound interferes with needed sugars on normal cells, too, with lethal results in animals. Now, Dutch researchers report that they've packaged the drug in nanoparticles targeted exclusively to cancer cells, and they've shown that this combination prevents cancer cells from metastasizing in mice.  

 is a web-based library of inquiry-based character-driven animated mysteries paired with differentiated supplemental activities that exposes students to the thrill of problem-solving. Mosa Mack allows educators to engage students while teaching standards-aligned content in a fun and engaging way while modeling scientific processing skills. Through its female protagonist whose passion for problem solving drives each story, Mosa Mack promotes diversity in the sciences while simultaneously providing teachers with an accessible way to incorporate inquiry into the classroom. Mosa Mack's unique inquiry-based approach targets the development of critical thinking skills for all students, with a particular focus on girls and minorities with historically low participation STEM fields.

The worst cancer cells don't sit still. Instead they metastasize-migrate from their original sites and establish new tumors in other parts of the body. Once a cancer spreads, it is harder to eliminate. A study by developmental biologists offers a fresh clue to how cancer cells acquire the ability to invade other tissues-a prerequisite for metastasis. It reveals that invasion requires cells to stop dividing. Therefore, the two processes- invasion and proliferation-are mutually exclusive. The finding could inform cancer therapies, which typically target rapidly proliferating cancer cells.

Panobinostat is a new type of drug that works by blocking an enzyme responsible for modifying DNA at the epigenetic level. Epigenetics refers to chemical marks on DNA itself or on the protein "spools" called histones that package DNA. These marks influence the activity of genes without changing the underlying sequence, essentially acting as volume knobs for genes. Earlier genomic studies showed that about 80 percent of DIPG tumors carry a mutation that alters a histone protein, resulting in changes to the way DNA is packaged and tagged with those chemical marks. This faulty epigenetic regulation results in activation of growth-promoting genes that should have been turned off, and shutdown of others that should have acted as brakes to cell multiplication. Cancer is the result. Panobinostat appears to work by restoring proper functioning of the cells' chemical tagging system.

This flipped case study explores how the topics of membrane structure, transport, and signaling via membrane-bound receptors are intimately associated with the paralysis of muscle targeted by botulinum neurotoxin. The case scenario revolves around a fictitious socialite that has requested the assistance of her personal concierge physician with a condition that has developed after having participated in a Hamptons Botox party. The physician and a shadowing pre-med undergraduate chat about the molecular mechanisms behind Botox induced muscle paralysis. The case is designed as an engaging capstone exercise for students to gain appreciation for how knowledge of basic cellular and molecular biology mechanisms are essential for pharmaceutical development and medical patient diagnosis and prognosis. Written for an undergraduate general biology course, the case is also suitable for use in courses such as cellular biology, neurobiology, or human physiology.

"This modular case study tells the story of Dan and Annie, a married couple of Acadian ancestry who have a genetic form of deafblindness called Usher syndrome. They live in Southwest Louisiana, home of the largest population of DeafBlind citizens in the United States. Acadian Usher syndrome is caused by an allele of the USH1C gene that came to Louisiana with the first Acadian settlers from Canada who founded today's Cajun population. This allele's single nucleotide substitution creates an erroneous splice site that produces a defective cytoskeletal protein (harmonin) of the cochlear and vestibular hair cells and retinal photoreceptors. This splice site is the target of a promising gene therapy. The case study applies and connects Mendelian inheritance, chromosomes, cell division, vision and hearing, DNA sequences, gene expression, gene therapy and population genetics to a specific gene and its movement through generations of Dan and Annie's families.  After the introduction, each of the remaining sections can be used independently either for in-class team activities or out-of-class extensions or assignments over an entire year of introductory undergraduate biology. "

In the study, published Monday (May 1) in Nature Biotechnology,  Luo and colleagues set their sights on two fusions genes they had previously found to be associated with prostate cancer and various forms of rapid and invasive cancer, including liver tumors. Using a modified CRISPR-Cas9 tool that creates a single- rather than double-stranded break in DNA, they targeted the chromosomal breakpoints that form these fusion genes and replaced fusion DNA with a gene encoding the enzyme HSV1-tk. This enzyme effectively kills tumor cells by converting the drug ganciclovir into its active form, which then blocks DNA synthesis and leads to cell death. (Ganciclovir is used to treat cytomegalovirus in humans.)

New immune system-boosting cancer drugs have in clinical trials saved the lives of many people with seemingly untreatable melanoma or lung cancer, but the drugs seem useless against colon cancer. One exception-a man with colon cancer whose metastatic tumors vanished for several years after he was treated in 2007-piqued researchers' interest. They suspected his recovery might have to do with the large number of mutations in his tumors. Now, a small clinical trial suggests that even cancer patients with types of tumors that were thought to be impervious to the new drugs could benefit if those malignancies have the right error-riddled DNA signature, a result that could help 3% to 4% of cancer patients.

n. As such, this set of Essential Elements addresses a small number of science standards, representing a breadth, but not depth, of coverage across the entire standards framework. The purpose of the DLM Essential Elements is to build a bridge from the content in the general education science framework to academic expectations for students with the most significant cognitive disabilities. This version of the Essential Elements will provide content for science assessments for at least the next two years. The DLM Science Consortium intends to develop a learning map based on research about how students learn science content and engage in scientific and engineering practices in the next phase of the project. Revisions will be made when the science map is complete, at which time we anticipate the EEs will be aligned to the map with revisions and additions as appropriate.