Pharmaceutical Elixir

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A Blueprint of Gateway Cloning

Gateway cloning is a method in which a vector of interest (plasmid=circular DNA) is recombined with an entry vector to obtain a new vector construct. This vector construct will bind to RBPs forming a complex that will bind to the RNA strand. This method is very useful for studying the functionality of RBPs.

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Remedies of Amyotrophic Lateral Sclerosis

Despite the fact that the cause of ALS remains unknown, there are numerous remedies for ALS to improve the quality of a patient's life. The pharmaceutical treatments are Riluzole, Baclofen, Diazepam, Trihexyphenidyl or Amitriptyline. Riluzole is a drug which slows down the progression of the disease. However, the drug cannot reconstruct the damaged motor neurons. Baclofen and Diazepam are drugs that will control the stiffness of muscle. Trihexyphenidyl and Amitriptylineare are prescribed to patients to control the flow saliva and phlegm. Besides medical remedies, there are also different forms of therapies such as physical, occupational, and speech which has helped to maintain the strength and endurance of muscles, to reduce pain levels, and to promote louder and clearer speech respectively.

A recent discovery by BrainStorm Cell Therapeutics in Israel reveals that adult stem cell therapy is able to stabilize the disease in some patients. The treatment proves effective as patients over a period of nine months show improvement in breathing, muscle strength, and speech. This clinical trial utilizes a patient’s bone marrow to transplant stem cells via treatment of the NurOwn stem cell technology. Currently, BrainStorm Cell Therapeutics is waiting for the approval of the FDA, so this treatment can also be offered to the public which will take place in the University of Massachusetts Medical School and Massachusetts General Hospital.

The patient’s stories on how stem cells have help them recovered from ALS provides potential hope for other patients. Not only does adult stem cell therapy work on ALS, it is also effective with cancer which can be cured by this type of treatment. The reason people are striving to promote this type is treatment is because it is a natural remedy that allows the body to repair damages without artificial substances. Stem Cells therapy is a useful remedy because it is safer and has more enhancing effects for the human body. 

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The Impact of RNA processing on Human Neurodegenerative Diseases (ALS)

As an undergraduate researcher in the Yeo Lab, I am currently studying the impact of RNA processing and the types of regulation that generates neurodegenerative diseases which occurs on the human gene. Majority of the time RNA sequences are always associated with RNA binding proteins (RBPs). RBPs recognize and bind to RNA elements within pre-mRNA sequence to form mRNA-protein (mRNP) complexes which regulates targeted RNAs.

The RBPs identify the RNA elements which control the functions and structures of the target RNA which constitute the mRNP code. This code is the basis for many human diseases; thus, any disruption in the code due to mutations in the RBPs or the RNA elements would result in abnormal phenotypes and neurodegenerative diseases such as ALS.

The RNA elements control the stability of RNA and regulate the process of translation of mRNA (messenger RNA is transcribed from DNA and serves as a blueprint in the synthesis of proteins). These elements either enhance or silence (or prevent) the process of converting the premature mRNA to the mature mRNA.

A recently study of 323 mouse RBPs via in situ hybridization, revealed that two-thirds of the RBPs studied were expressed in the brain. This study implies that neurons depend heavily on RBPs to function properly. In the nervous system, the binding of RBPs to the mRNA acts as a shield which protects the mRNA to properly synthesize proteins. RBPs that ensure the stability of the mRNA play an important role in the process of learning and memory. This suggests that RBPs are the building blocks in maintaining balance and stability in the nervous system.

In order to develop a realistic and effective disease models for ALS, is it crucial to acquire an understand of how RBPs control RNA processing. The first step is to discover the various binding activities between RNA elements and RBPs. The second step is to understand the mRNP codes that corresponds to differentiation, development, and survival of neural stem cells. The information acquired from these first two steps will provide the necessary information in order to relate the defects in RNA processing to neurodegenerative diseases like ALS. By grasping the knowledge of the causes of neurodegenerative diseases at a microscopic level, the pharmaceutical industry can utilize this information to develop medication to find a cure for these neurodegenerative diseases.

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The Causes of Amyotrophic Lateral Sclerosis (ALS)

When I was a volunteer at the San Clemente, the first patient who I encountered was Grandpa Boris. He was a being filled with love and life which brightened my day. Unfortunately, behind all of his smiles, he was suffering from Amyotrophic Lateral Sclerosis (ALS). Tragically, his enthusiasm and energy diminished day by day as his symptoms for ALS continued to worsen.

Eventually, Grandpa Boris lost the ability to move and soon also lost his ability to communicate. He could no longer play with his grandchildren who came to visit him on Sundays. He sat helplessly in his wheelchair as his sad and dismal eyes gazed upon his grandchildren’s playful nature. Seeing his deterioration saddens me because I knew him as a happy and delightful individual. At the time, I felt frustrated because I was not able to help Grandpa Boris since I did not have the proper education or training. From my time working at the hospital, I have learned that it is important to know the pain of patients; however, it is more crucial to understand the causes of the pain.

The symptoms that Grandpa Boris suffered were caused by the progressive deterioration of motor activities that regulates muscle movements and flexing of muscles. ALS also known as Lou Gehrig’s Disease occurs when the motor neurons are slowly diminishing; hence, resulting in muscle paralysis. This occurrence will interfere with the networking between the brain and the spinal cord. The malfunctioning between the brain and the spinal cord will result in the muscles losing strength and slowly losing its function to carry out actions that require physical abilities.

Although there is not a primary cause of ALS, there are many postulating explanations. The various investigated causes of ALS inclusively include inherited genetic mutations, head trauma, military service, and sports that require physical contact, chemical exposures, electromagnetic field exposures, and viral infections. One theory that explains the destruction of motor neurons involves glutamate, a chemical that transmits messages between the brain and nerve cells. When glutamate accumulates between the synapses, a high influx of calcium will enter the motor neurons resulting in the destruction of the nerve cells. A second theory for the damage of nerve cells is a mutation in the gene that creates superoxide dismutase, an enzyme that protects the body from damages produced by toxic free radicals. ALS patient may have this abnormal gene; thus, when free radicals build up in the body, the cell will undergo cell death (apoptosis). Ultimately, these highly reactive molecules will destroy nuclear DNA and proteins and lead to the degeneration of the motor neuron.

Due to the insufficient knowledge of what causes ALS, there are numerous ongoing research projects searching for a possible explanation for the death of the motor neuron. Once the cause has been identified, drugs will be produced in order to treat ALS. The current research projects will grant ALS patients, such as Grandpa Boris, a future in which he can feel the warm embrace of his grandchildren.