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Transcript of MEDICAL/PHARMACEUTICAL BIOTECHNOLOGY
By Julia, Samantha, Tara and Gelila
"a field that uses microorganisms, macroorganisms and hybridomas to create pharmaceuticals that are safer and more cost effective than conventionally produced pharmaceuticals"
Branches of Research
medicines from plants, animals, fungi and genetically engineered cells
monoclonal and polyclonal antibodies
vaccine and gene therapy
prosthetics, artificial or engineered organs and tissues
designer drugs and antibodies
The treatment of inherited diseases by corrective genetic engineering of the dysfunctional genes
It is part of a broader field called genetic medicine
Involves the screening, diagnosis, prevention and treatment of hereditary conditions in humans
Results of genetic screening can pinpoint a potential problem to which gene therapy can sometimes offer a solution
What is the purpose?
Used to correct a genetic defect
Used to reduce the symptoms of a genetically-determined disease when the introduced gene is expressed and its product is produced
Pharmaceutical companies use biotechnology as a way to manufacture drugs, gene therapy, and genetic testing. Biotech companies make biotechnology products by manipulating and modifying organisms, typically at molecular level.
In 1999, scientists had isolated Atonal . Atonal is a gene which acts as a switch to turn on the growth of inner ear hair cells, untimately carrying sound waves and translating them into electrical signals in the brain.
Atonal Gene Therapy for Hearing Loss
Humans are born with hair cells but the atonal switch flips off at birth
After this discovery, a biotech company called GenVec came up with a way to flip the switch back on.
GenVec used a viral vector. Viral Vector is a virus that has been modified making it harmless so that it can carry the atonal gene.
The viral vector carries the gene into the cells that line the insides of the cochlea, some of which can turn into hair cells.
Within the cells, atonal starts to switch the flip back on and the newly formed hair cells wire themselves into the auditory cortex also known as the part of the brain that processes sound.
GenVec researchers displayed in 2005 that their vector carrying the mouse version of the gene could regenerate hearing in deafened rodents.
During 2011, scientists showed that this drug also restored balance in rodents.
In addition, this drug must only be taken once in order to begin regeneration of hearing loss.
Also known as Plant-made pharmaceuticals
The production and use of transgenic (contains genes from a different plant or animal that have been introduced using a laboratory technique) plants and animals genetically engineered to produce medical substances for the use in humans or animals
Genetic engineering techniques used to induce crops such as corn, tomatoes, and tobacco, producing high concentrations of high-value pharmaceuticals
The process of developing and using plants to produce pharmaceutical compounds consists of identifying a target protein and then identifying and isolating the gene that codes for the protein
Pharmaceutical crops lower production and capital costs (their greater production flexibility give them a strong appeal as biofactories for drug development)
why is it used?
Number of new biopharming drugs and vaccines legalized (graph stops at 2002, however it can be inferred the increase continued)
'Biopharming' Offers A Powerful New Approach To Ebola And Other Diseases
The use of plants to produce life-saving pharmaceuticals captured global attention when the Ebola drug"ZMapp" was revealed
ZMapp is produced in the leaves of tobacco plants.
The ZMapp vaccine which comes from plants is planed to be faster and cheaper than the previous methods which use chicken eggs to grow the virus needed to make the vaccines.
ZMapp is currently being tested on elderly people, and plans to launch a large human trial in 2016.
ZMapp is scheduled to hit the market in 2019
ZMapp is made of three Ebola antibodies, two of which were produced at Canada’s National Microbiology Laboratory in Winnipeg.
ZMapp, which has been given to a number of people who were infected with Ebola had infact survived , showing a remarkable recovery after receiving the treatment.
Biopharming drug discovery process.
an artificial body part
a man-made device that is implanted into a human to replace a natural organ
purpose is to restore a specific function
a machine, device, or other material that is used to substitute the functions of a missing part of the human body.
Due to biotechnologies advanced research on prosthetic limbs, amputees are able to receive a prosthesis that has a direct connection to the bone, nerves and muscles
The robotic prostheses is controlled by an implanted neuromuscular interface.
First, a titanium implant is surgically inserted into the bone where, over time, it becomes fixated to the bone in a process called osseointegration.
Then, a metallic extension is connected to the implant.
The robotic prosthesis connects to the living arm
Electrodes that are implanted in the nerves and muscles help control the prosthetic.
These electrodes record touch signals that are transmitted through the implant to the brain.
The electrodes can pick up more muscle and nerve signals than electrodes on the skin.
The patient can control the prosthesis with less effort than before.
Maneuvering the robotic arm is also more precise, so the patient can handle small and delicate items.
Overall look at
MEDICAL AND PHARMACEUTICAL
Benifit our lives ultimately by meeting the human needs and demands in order to improve our quality of life.
By manufacture drugs such as ZMapp, gene therapy, and creating life altaring prothetics, medical/pharmaceutical biotechnology evidently impacts human society in a positive way.
Engineered Epstein-Barr (EBR) virus
– specific Cytotoxic T lymphocytes to express a chimeric antigen receptor directed to a
They reason that these genetically engineered lymphocytes would receive optimal constimulation after engagement of the original receptors
Cytotoxic T lympthocytes
: type of white blood cell that plays a central role in cell-medicated immunity
substance capable of inducing a specific immune response
Chimeric antigen receptor
: artificial T cell receptors – engineered receptors, which graft an arbitrary specificity onto a regular t-cell
T cell immunotherapy extended into clinical application
The benefits of T cells are being expanded by engineering T lymphocytes to express chimeric antigen receptors (CARs) that are able to recognize specific antigens expressed on the cell surface of different types of tumor cells
Researchers designed a clinical study in which patients with non-Hodgkin lymphomas (NHLs) were infused with 2 autologous T cell products
As researchers enrolled 6 patients, with relapsed or refractory NHL, each patient had the active disease
They generated 2 CAR-transduced T cell products for each patient, always from the same blood collection.
All individuals received the T cell products at least 6 weeks after their last chemotherapy treatment.
At the time of infusion, patients had measurable disease by imaging studies (CT or PET scan) or by physical examination
T cell products were administered simultaneously to each patient
Second infusions were allowed if there was evidence of clinical benefit at 6 weeks after the first infusion
Researchers assessed toxicity on the basis of patient interviews, physical examinations, and laboratory tests of organ function at 1, 2, 4, and 6 weeks and 3, 6, 9, and 12 months after infusion. (Ultimately noticed a positive result)
MicroRNAs are small, RNA molecules encoded in the genomes of plants and animals.
These highly conserved RNAs regulate the expression of genes by binding to the 3 untranslated regions of specific mRNAs.
A microRNA (abbreviated miRNA) is a small non-coding RNA molecule (containing about 22 nucleotides) that functions in RNAsilencing and post-transcriptional regulation of gene expression.
MicroRNAs are produced from either their own genes or from introns.
In the last two to three years has the importance and diversity of this class of small, regulatory RNAs been appreciated.
Each miRNA is thought to regulate multiple genes, and since hundreds of miRNA genes are predicted to be present in higher eukaryotes, the potential regulatory circuitry afforded by miRNA is enormous.
Several research groups have provided evidence that miRNAs may act as key regulators of processes as diverse as early development, cell proliferation and cell death, apoptosis and fat metabolism, and cell differentiation
The Present and The Future
Recent studies of miRNA expression implicate miRNAs in brain development, chronic lymphocytic leukemia , colonic adenocarcinoma, Burkitt’s Lymphoma, and viral infection
Researchers are suggesting possible links between miRNAs and viral disease, neurodevelopment, and cancer.
MicroRNAs in liver tissue engineering - New promises for failing organs
Increased knowledge of tissue-selective microRNA expression now allows the engineering of replicating deadly viruses and diseases.
The engineered viruses contain engineered microRNA that will fight against the specific virus/disease.
MicroRNA RNA fragments withing the engineered viruses are being created to prevent the production of a particular protein by binding to and destroying the messenger RNA that would have produced the protein.
Ex. cancer cells contain certain proteins within them that aid in the rapid production of the cells.
Biomedical engineers have been experimenting with the microRNA in these cells and 'recoding' them to prevent these proteins from being created.
Researchers are testing this theory with animals to try and fix liver toxicity and repair the damaged liver.
Researchers say that for cancer this is a much less harmful way to prevent or slow cancer cells than chemotherapy.
Believe that they will have the ability to only be repairing specific tissues in select organs.
Researchers are looking into creating vaccine type formulations of these viruses in order to help people.
Researchers have found that microRNAs may serve as therapeutic targets of cancer’s spread from its initial site to other parts of the body. In this image, breast cancer cells (right) spread toward the hindlimb bone (left), using the host's own bone-destroying cells. The tumor uses the osteoclasts as forced labor however MicroRNAs can reduce that forced labor by restraining the osteoclast proteins, therefore limiting the number of osteoclasts present.
Proposed plan for the treatment of liver cancer with combined chemotherapy and miRNA-based therapy.
At this point the best available chemotherapeutic option could be combined with miRNA-based therapy
Potential patients assessed by measuring circulating miRNAs in patient serum or tumoral miRNAs from a biopsy.
After treatment, the patient could be checked for relapse by periodically studying circulating miRNAs from serum. The presence of miR-21 could indicate a potential relapse, and treatment would resume
In miRNA profiling, those present in the tumour, such as miR-21, could be restricted. Suppressor miRNAs cells decreases the quantity of the cellular component in the tumour that could be restored and miR-26 levels could be increased with miRNA mimics
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