סמינריון בוגר- הנחיות למורים ולתלמידים:FROM THE UNIVERCITY Graduate seminar - instructions for teachers and students:Student guide for graduate seminar work:• The length of the work is 5...

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nin- viral gene therapy in the CNS(nanomedicine)


סמינריון בוגר- הנחיות למורים ולתלמידים: FROM THE UNIVERCITY Graduate seminar - instructions for teachers and students: Student guide for graduate seminar work: • The length of the work is 5 single-spaced pages, not including the list of cited literature. • The order of the chapters in the seminar will be as follows: • Introduction - methods - results - discussion - conclusions - literature list • A list of references must be written according to the convention in the field of research in which he is engaged the seminary. • For example: Arnesano, F.; Scintilla, S.; Natile, G., Interaction between platinum complexes and a methionine motif found in copper transport proteins. Angew. Chemie - International Edition 2007, 46, (47), 9062-9064. Title of seminar: Non-viral gene therapy in the CNS (central nervous system) MY NOTES Introduction 1 or 2 pages 1.what is gene therapy in general (you can take this general information from the article gene therapy , viral pdf + Non Viral Vectors in Gene Therapy- An Overview NOT NECCERLY NEED TO USE THE WHOLE ARTICLE – YOU CAN TAKE WHAT IS NEEDED 2.viral virus vs. non-viral (the advantages and disadvantages of both – and why is non-viral virus delivery is better that the viral virus delivery at gene therapy (you can take this general information from the article gene therapy ,viralpdf + Non Viral Vectors in Gene Therapy- An Overview also its written in Nanodiamond….. ) main article for this work assignment) or for viral vectors you can use this paragraph or just use from the main article(my seminar work about the non-viral. Also, I need to mention “shortly “the viral vector delivery and why the non viral is better – and the you can write about the non- viral extensively) Viral vector is the most effective means of gene transfer to modify specific cell type or tissue and can be manipulated to express therapeutic genes. Several virus types are currently being investigated for use to deliver genes to cells to provide either transient or permanent transgene expression. These include adenoviruses (Ads), retroviruses (γ-retroviruses and lentiviruses), poxviruses, adeno-associated viruses, baculoviruses, and herpes simplex viruses. The choice of virus for routine clinical use will depend on the efficiency of transgene expression, ease of production, safety, toxicity, and stability. This chapter provides an introductory overview of the general characteristics of viral vectors commonly used in gene transfer and their advantages and disadvantages for gene therapy use. 3.to continue writing about -non viral gene therapy in general (because the assignment about it) you can use the articles that I’ve mentioned it before and this article Non-viral gene therapy: Gains and challenges of non-invasive administration methods - methods - results – discussion (3or 4 pages)- the most important thing is to write about the research article 4. to talk about one methods of non-viral gene therapy in the CNS(I’ve found an article about Nanodiamond -cationic noisome they used this method to deliver to the CNS by the non-viral. )main article( you can use this article)main article(Nanodiamond Integration into Niosomes as an Emerging and Efficient Gene Therapy Nanoplatform for Central Nervous System Diseases - its important to read and write about this article and to take all of the informations - but the other articles you can take the information that you need - and conclusion of the research article and non viral gene delivery in theCNS I think that will help you to understand the assignment more:- this picture is about--- ,a delivery methods (viral and non-viral ) to deliver DNA into the CNS I’ve found(not given by the teacher- its self-work) those article that I mentioned above . the most important thing the order of the assignment Introduction - methods - results - discussion - conclusions - literature list that the university request WHICH YOU CAN USE MAIN ARTICLE Nanodiamond Integration into Niosomes as an Emerging and Efficient Gene Therapy Nanoplatform for Central Nervous System Diseases This work-assignment is important to me so – if you don’t understand - please feel free to ask me ….
Answered 8 days AfterDec 16, 2022

Answer To: סמינריון בוגר- הנחיות למורים ולתלמידים:FROM THE UNIVERCITY Graduate seminar - instructions for...

Dr Insiyah R. answered on Dec 19 2022
30 Votes
Introduction    2
Method    2
Results    2
Discussion    3
Limitations    4
Conclusion    4
Reference    6
Introduction
The process of genetically altering the cells of a patient in order to enhance their health is known as gene therapy. An innovative approach to treating both inherited and acquired disorders involves inserting therapeutic gene information and its associated regulatory mec
hanisms into the nucleus. This allows for the correction of mutation-induced loss of function or the expression of the defective gene product at physiological levels (Foldvari et a,2016). It is well-known that almost all human illnesses are brought on by a lack of one gene or a mutated combination of genes. There are two approaches to carrying out CNS gene transfer in living creatures: in vivo, where the gene-vector conjugate is immediately injected further into the animal; and ex vivo, in which the desired gene is first transfected into neuronal cell culture before being reintroduced into the animal (Ramamoorth & Narvekar,2015). The scientific community has speculated for more than 50 years that treatments based on the transmission of genetic elements would be a desirable choice to combat human illnesses. Theoretically, this approach—so-called gene therapy—would provide the opportunity to achieve a long-lasting and curative therapeutic effect. This method is often used in clinical trials, and some have only recently been given the status of authorised medications in the United States and Europe (Al Qtaish et al,2022). To provide a more accurate and translational milieu for an in vivo model, further gene delivery studies using nano-duplexes at a 5/1 lipid/DNA ratio in primary CNS cells from cerebral and retinal sources have been conducted. The immunocytochemistry detected the GFP signal in both primary cell cultures, demonstrating the ability of nano-duplexes to transfer genetic material to CNS cells successfully. Additionally, it is likely that NDs, rather than other carbon-based nanomaterials, would have a better correlation with biological systems, making them a promising contender for biomedical applications (Al Qtaish et al,2022). The discovery that NDs play a growing function in niosomes for gene delivery applications is significant since CNS illnesses represent a field where the creation of novel treatment approaches represents a critical need. The non-viral techniques for in vivo gene transfer in the CNS will be the main emphasis of this review. This assignment will explain the non-viral gene therapy in the CNS in detail.
Method
In order to achieve the precise tomographic tilt series alignment needed, a 1 mg/mL sample was diluted to 0.5 mg/mL using bovine serum albumin (BSA) gold nanoparticles (10 nm). Following vortex shaking, 3 L of the sample was placed to the Cu/ Rh R2/2 Quantifoil grid and vitrified at 22 °C with 95% humidity using a ThermoFisher Scientific Vitrobot Mark IV (Al Qtaish et al,2022). Utilising ThermoFisher Scientific's Tomography programme (step 3°, "65° at 28.000," with a pixel size of 1.44 nm/pix), tilted series were acquired to an actual figure dose of 130 e- 2 utilising the dose-symmetric tilt method. Utilising IMOD, the tilt series synchronisation was carried out, evaluated, and rendered in three dimensions using TOMO3D and SIRT reconstruction. Rebuilt volumes for USFC Chimera (Al Qtaish et al,2022).
Results
Compared to nioplexes, nanodiaplexes had greater levels of endocytosis via clathrins (p 0.05), higher lysosomal colocalisation (p 0.05), and endosomal escape characteristics. However, endocytosis mediated by caveolae was the most effective mechanism in the case of nanodiaplexes (Al Qtaish et al,2022). Nanodiaplexes successfully transfected retinal and neuronal cells, according to investigations in CNS primary cells...
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