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Cell and gene therapy future prospective

Feb 24, 2024 | Science, Technology

Cell and gene therapies represent revolutionary approaches in the field of medicine, offering promising avenues for treating a myriad of diseases at their core genetic and cellular levels. With rapid advancements in biotechnology and our deepening understanding of genetics, the future of healthcare is poised to be transformed by these innovative therapies. This essay explores the future prospects of cell and gene therapy, envisioning a landscape where personalised medicine becomes the norm, and previously untreatable conditions are conquered.

History

The history of cell and gene therapy is a fascinating journey marked by significant scientific breakthroughs, setbacks, and remarkable advancements. Here’s an overview of key milestones in the development of these innovative therapies:

Early Exploration (1970s-1980s):

In the 1970s, the groundwork for cell and gene therapy was laid through foundational discoveries and early experiments that set the stage for future advancements in the field. While the term “gene therapy” had not yet been coined, researchers began to explore the potential of manipulating genetic material for therapeutic purposes. Here are some notable events and developments from the 1970s that contributed to the history of cell and gene therapy,

Hamilton O. Smith and Kent W. Wilcox (1970) discovered the first restriction enzyme, HindII, while studying the bacterial immune system. This breakthrough paved the way for the manipulation and analysis of DNA, which would later become instrumental in genetic engineering techniques used in gene therapy. In 1971, Werner Arber, Daniel Nathans, and Hamilton O. Smith independently isolated the first restriction enzyme, HindII (later renamed HindIII), from the bacterium Haemophilus influenzae. This enzyme could specifically cut DNA at particular sequences, enabling scientists to manipulate DNA for various applications, including gene cloning and sequencing.

Arthur Kornberg and colleagues discovered DNA ligase in 1971, an enzyme capable of joining together DNA fragments. DNA ligase played a crucial role in early genetic engineering experiments by allowing researchers to insert foreign DNA into plasmids, a key step in recombinant DNA technology.

In 1972, Theodore Friedmann and Richard Roblin published a seminal paper in the journal Science titled “Gene Therapy for Human Genetic Disease?” In this paper, they proposed the concept of using genetic material to treat inherited diseases, laying the theoretical foundation for gene therapy. The development of recombinant DNA technology by researchers like Paul Berg who successfully combined DNA from two different organisms, and seminal work by Stanley Cohen, and Herbert Boyer in the early 1970s paved the way for gene cloning and the creation of genetically engineered organisms, laying the groundwork for future gene therapy approaches.

The United Kingdom also played a significant role in the early development of cell and gene therapy, particularly through pioneering research and scientific contributions that laid the groundwork for future advancements in the field In 1973, the University of Edinburgh hosted the landmark Gordon Conference on Nucleic Acids, where scientists discussed recent advances in molecular genetics, including the emerging field of recombinant DNA technology. Scientists like Richard Roberts and Phillip Sharp at the University of Cambridge made key discoveries related to the splicing of RNA, which would later have implications for gene therapy approaches targeting genetic diseases.

As genetic research progressed, researched in the UK started the, discussions about the ethical implications of genetic engineering and potential risks associated with gene therapy interventions began to emerge, laying the groundwork for the establishment of ethical guidelines and regulatory frameworks in subsequent decades.

The first gene therapy trial was conducted in 1980 by Martin Cline, who attempted to treat a patient with a genetic blood disorder called beta-thalassemia using genetically modified cells. Although the trial was unsuccessful, it paved the way for future research in the field.

While the 1970s primarily laid the groundwork for the field of gene therapy through foundational discoveries and theoretical proposals, it wasn’t until the following decades that experimental research and clinical trials began in earnest, leading to the development of the first gene therapy techniques and treatments.

Development of Viral Vectors (1980s-1990s):

During the 1980s, significant progress was made in the field of cell and gene therapy, laying the foundation for future clinical applications and advancements. Here are some key events and developments from that decade:

The development of viral vectors, such as retroviruses and adenoviruses, revolutionised gene therapy by providing efficient delivery systems for introducing therapeutic genes into target cells. Retroviruses, such as murine leukemia virus (MLV), were modified to serve as vectors capable of efficiently delivering therapeutic genes into target cells. These advancements paved the way for future gene therapy trials using viral vectors. Also, the term “gene therapy” was first coined in the 1980s to describe the concept of using genetic material to treat or cure diseases. Scientists and clinicians began to recognize the potential of gene therapy as a novel approach to treating genetic disorders, cancer, and other diseases at their root genetic causes.

Furthermore, in 1980s, researchers achieved significant breakthroughs in the creation of transgenic animals, which carried foreign genes deliberately inserted into their genomes. These genetically modified animals served as valuable models for studying gene function, disease mechanisms, and potential gene therapy approaches. As gene therapy research progressed, ethical and regulatory considerations became increasingly important. Scientists and policymakers engaged in discussions about the potential risks and benefits of gene therapy, as well as the ethical implications of manipulating human genetic material. These discussions laid the groundwork for the development of ethical guidelines and regulatory frameworks governing gene therapy research and clinical trials.

While PCR was invented in the United States in the 1980s by Kary Mullis, its widespread adoption and further refinement involved contributions from scientists worldwide, including those from the UK. British researchers played important roles in optimising PCR protocols, developing new PCR reagents, and applying PCR techniques to various fields of research. The development of PCR significantly facilitated gene cloning and amplification efforts. British scientists were instrumental in the development of plasmid cloning vectors, which are circular DNA molecules that can replicate independently of the host genome. Plasmids served as essential tools for cloning and amplifying genes of interest. Research groups led by scientists like Sydney Brenner and Richard Durbin at the MRC Laboratory of Molecular Biology in Cambridge contributed to the development of plasmid cloning vectors.

In 1990, the first successful gene therapy trial was reported by W. French Anderson and his collaborative team of British researchers, who treated a patient with severe combined immunodeficiency (SCID) using a retroviral vector to deliver a functional copy of the defective gene.

George Dickson, a molecular biologist, conducted research in the 1980s that contributed to the development of gene therapy strategies for muscular dystrophy. His work focused on the delivery of therapeutic genes to muscle cells using viral vectors, laying the groundwork for future preclinical and clinical studies in the field.

Overall, the 1980s were a formative period in the history of cell and gene therapy, marked by pioneering research, technological advancements, and the birth of a new field aimed at harnessing the power of genetics to treat human diseases. While significant challenges remained, including safety concerns and technical limitations, the groundwork laid during this decade paved the way for future progress and clinical applications in gene therapy.

What is CCD?

Aug 17, 2020 | Technology

CCD (Charge-Coupled Device) and CMOS (Complementary Metal-Oxide-Semiconductor) are two distinct types of image sensors used in digital cameras, smartphones, and various imaging devices. While both technologies serve the same purpose of converting light into electrical signals for capturing images, they have significant differences in their working principles, performance, and applications. Here are some of the key differences between CCD and CMOS image sensors: Charge-Coupled Devices (CCDs) represent a breakthrough technology that has profoundly impacted various fields, from photography and astronomy to scientific research and digital imaging. Developed in the late 1960s, CCDs have revolutionized the way we capture, store, and process visual information.

The Working Principle of CCDs:
A Charge-Coupled Device is a semiconductor device designed to detect and convert light into electrical signals, which can be processed and transformed into digital images. The heart of a CCD is an array of photosensitive elements, often referred to as pixels. Each pixel consists of a photosensitive region, typically made of silicon, capable of accumulating and storing electric charge when exposed to light.

The process of converting light into electrical charge begins when photons strike the photosensitive region of the pixel, causing the release of electrons. These freed electrons are then accumulated in potential wells formed within the silicon structure. When the image acquisition process is complete, the accumulated charge is transferred, one row at a time, to an adjacent shift register. This process of charge transfer continues until the electrical signals are read out and processed to generate a digital image.

CMOS: CMOS image sensors, on the other hand, use a different approach. Each pixel in a CMOS sensor has its own individual amplifier, allowing the conversion of photons into electrical charge and the amplification of the signal to happen within each pixel. This parallel readout approach is different from the serial transfer used in CCDs.

Power Consumption:

CCD: CCD sensors tend to consume more power compared to CMOS sensors. This is because of the complex charge transfer process, which requires high-voltage clocks and multiple capacitors for each pixel.

CMOS: CMOS sensors are generally more power-efficient since they employ individual amplifiers at each pixel, reducing the need for high-voltage clocks and lowering power consumption.

Noise Performance:

CCD: CCDs traditionally have lower noise levels due to their design and analog nature of signal processing. This makes CCD sensors well-suited for applications that require high-quality, low-noise imaging, such as professional photography and astronomy.

CMOS: Modern CMOS sensors have made significant advancements in noise reduction and can now offer noise levels comparable to CCDs. However, in certain high-end applications, CCDs may still have a slight advantage in noise performance.

Speed and Frame Rates:

CCD: CCD sensors have historically been preferred for applications that require high image quality and relatively lower frame rates, such as studio photography or scientific imaging.

CMOS: CMOS sensors have improved over time and now offer faster frame rates, making them more suitable for applications like video recording, sports photography, and real-time imaging.

Manufacturing Complexity and Cost:

CCD: The manufacturing process for CCD sensors is more complex and expensive compared to CMOS sensors. This complexity contributes to the higher cost of CCD-based imaging devices.

CMOS: CMOS sensors are less complex to manufacture, making them more cost-effective for mass production. As a result, CMOS-based devices, such as smartphones and consumer digital cameras, are more prevalent and affordable.

Applications of CCDs:

Photography: CCDs have significantly enhanced the quality of photography, replacing traditional film cameras with digital cameras. The ability to capture images digitally offers several advantages, including immediate image preview, easy storage, and post-processing capabilities. Digital cameras with CCD sensors have become ubiquitous in today’s world, empowering individuals to capture and share visual experiences effortlessly.

Astronomy: CCDs have revolutionized the field of astronomy by enabling sensitive and precise image capture of celestial objects. Their high sensitivity and low noise characteristics make CCDs indispensable tools for capturing faint light from distant stars and galaxies. They are extensively used in space telescopes, such as the Hubble Space Telescope, and ground-based observatories, aiding astronomers in their quest to unlock the mysteries of the cosmos.

Scientific Research: CCDs have found extensive applications in scientific research, allowing researchers to visualize and analyze a wide range of phenomena. In fields like biology, microscopy using CCDs enables the study of cellular structures and processes at high resolutions. In physics, CCDs play a vital role in particle detectors, capturing the trajectories of subatomic particles with remarkable precision.

Security and Surveillance: CCD-based cameras are commonly used for security and surveillance purposes. Their ability to provide clear, high-resolution images, even in low-light conditions, makes them ideal for monitoring and safeguarding various environments, from residential areas to high-security facilities.

The Advantages and Limitations of CCDs:
CCDs offer numerous advantages, such as high sensitivity, low noise, and excellent spatial resolution. These qualities make them highly desirable for applications requiring detailed imaging and accurate data acquisition. Additionally, CCDs exhibit high quantum efficiency, meaning they can efficiently convert incoming photons into electrical signals.

However, CCDs are not without limitations. One major drawback is their susceptibility to blooming, a phenomenon in which overexposure of bright light sources can cause charge to spill into adjacent pixels, resulting in streaks and inaccuracies in the image. Moreover, CCDs consume more power than some other imaging technologies, and they can suffer from charge transfer inefficiencies and dark current noise.

Conclusion:
Charge-Coupled Devices have revolutionized the world of imaging and visual data acquisition, becoming an integral part of modern technology in photography, astronomy, scientific research, and security applications. Their ability to accurately capture and convert light into electrical signals has opened up new horizons in various fields of study and everyday life. As imaging technology continues to evolve, CCDs remain a foundational technology, inspiring further advancements in digital imaging and ensuring that the world continues to see through ever-clearer lenses.

SPR (Surface Plasmon Resonance) technology

May 23, 2020 | Technology

SPR (Surface Plasmon Resonance) technology is a powerful and versatile analytical technique used to study molecular interactions in real-time. It is widely employed in various scientific fields, including biochemistry, biophysics, pharmaceutical research, and environmental monitoring. SPR technology provides valuable insights into the kinetics, affinity, and specificity of biomolecular interactions, offering a deeper understanding of complex biological processes and aiding in drug development and diagnostics.

Working Principle:
SPR is based on the principle of the excitation of surface plasmons, which are collective oscillations of free electrons that occur at the interface between a thin metal film (usually gold or silver) and a dielectric medium (typically glass or prism). When polarized light, such as laser light, is directed onto the metal surface at a specific angle known as the resonance angle, surface plasmons are excited, leading to a decrease in reflected light intensity.

When molecules bind to the metal surface, the local refractive index changes, altering the resonance condition and causing a shift in the angle of minimum light reflection. By monitoring this shift in real-time, researchers can quantitatively measure the interactions between molecules immobilized on the metal surface and those present in the surrounding solution.

Application in Biomedical Research:
SPR technology is widely used in the field of biomedical research to investigate a variety of biomolecular interactions. Some of the key applications include:

Protein-Protein Interactions: Researchers can study the binding interactions between proteins, determining their kinetics, affinities, and specificity. This information is crucial for understanding signaling pathways, enzymatic reactions, and protein-protein interactions in disease processes.

Protein-Ligand Interactions: SPR is valuable in drug discovery, as it allows the screening and characterization of potential drug candidates binding to target proteins. This aids in identifying molecules with high binding affinities, which may serve as leads for drug development.

Antibody-Antigen Interactions: SPR is used extensively in immunology to investigate antibody-antigen interactions, enabling the study of immune responses, antibody specificity, and the development of diagnostic assays.

DNA-Protein Interactions: SPR can be employed to examine the binding interactions between DNA and proteins, providing insights into transcription factor binding, DNA repair processes, and chromatin remodeling.

Advantages of SPR Technology:
SPR offers several advantages over traditional methods for studying molecular interactions:

Real-Time Analysis: SPR provides real-time, label-free measurements of molecular interactions, eliminating the need for fluorescent or radioactive labeling of molecules.

High Sensitivity: SPR can detect minute changes in molecular interactions, allowing for the study of weakly interacting or low-concentration molecules.

No Requirement for Conformational Changes: Unlike other techniques, SPR does not require conformational changes in molecules for detection, enabling the study of molecules in their native state.

Quantitative Analysis: SPR provides quantitative data on binding kinetics (association and dissociation rates) and equilibrium dissociation constants (KD), facilitating precise characterization of interactions.

Conclusion:
Surface Plasmon Resonance (SPR) technology has emerged as a powerful tool for studying molecular interactions in real-time. Its wide-ranging applications in biomedical research, drug development, and diagnostics have significantly contributed to our understanding of complex biological processes. By offering label-free, real-time, and quantitative data, SPR technology continues to be a valuable asset in unraveling the intricacies of molecular interactions and advancing scientific knowledge across various disciplines.

Molecular biology

May 18, 2020 | Science, Technology

Molecular biology is a discipline that has revolutionised our understanding of life at the most fundamental level. By investigating the interactions and processes that occur within biological molecules, molecular biologists have unveiled the intricate mechanisms governing the structure, function, and regulation of cells.

The field of molecular biology emerged in the mid-20th century as a multidisciplinary science that explores the biological phenomena at the molecular scale. It unites principles from genetics, biochemistry, and cell biology to decipher the blueprint of life encoded within the DNA, and how it directs the cell’s activities to maintain life and perpetuate genetic information. By investigating the interplay of biomolecules, molecular biologists have provided deep insights into the complex mechanisms that govern living organisms.

Historical Developments:

The historic foundation to molecular biology originated in the 1930s and 40s, making it a relatively new scientific field and arose from the convergence of work by geneticists, physicists, and structural chemists on a common problem: the nature of inheritance. Warren Weaver, director of the Division of Natural Sciences at the Rockefeller Foundation, coined the term “molecular biology” in 1938 in a report for the Foundation. Weaver and others encouraged research at the intersection of biology, chemistry and physics, while prominent physicists such as Niels Bohr and Erwin Schrödinger turned their attention to biology to explain the biochemical and biophysical basis of life. For Schroedinger, biology was to be reduced to the more fundamental principles of physics, while Max Delbrueck instead resisted such a reduction and sought what made biology unique. The former student of Thomas Morgan’s, Hermann J. Muller shift from Mendelian genetics to the study of gene structure raised the question of the relation between the gene concepts found in those separate fields of genetics. And the import of experimental methods from physics to biology raised the question of the relation between those disciplines. Also, during the period between 1930s to 40s, other emerging cross-disciplinary fields such as colloid chemistry, biophysics and radiation biology, crystallography, have aided the development of molecular biology as a field of it’s own right.

The formal foundations of molecular biology were laid with the discovery of the DNA double helix structure by Watson and Crick in 1953. This breakthrough unlocked the key to understanding how genetic information is stored and replicated. Subsequent advances in molecular biology, such as the elucidation of the genetic code and the discovery of DNA polymerases and RNA polymerases, paved the way for molecular genetics.

In the 1970s, the development of recombinant DNA technology, commonly known as genetic engineering, enabled scientists to manipulate and transfer genetic material between different organisms. This revolutionary technique has since had a profound impact on various fields, from agriculture to medicine.

The discoverers of the structure of DNA. James Watson (b.1928) at left and Francis Crick (b.1916), with their model of part of a DNA molecule in 1953. Crick and Watson met at the Cavendish Laboratory, Cambridge, in 1951. Their work on the structure of DNA was performed with a knowledge of Chargaff’s ratios of the bases in DNA and some access to the X-ray crystallography of Maurice Wilkins and Rosalind Franklin at King’s College London. Combining all of this work led to the deduction that DNA exists as a double helix. Crick, Watson and Wilkins shared the 1962 Nobel Prize for Physiology or Medicine, Franklin having died of cancer in 1958. Credit: A. Barrington Brown / Photo Researchers, Inc..

The Central Dogma of Molecular Biology: Decoding Life’s Blueprint

The Central Dogma of Molecular Biology is a foundational principle that underpins our understanding of the flow of genetic information within living organisms. Coined by Nobel laureate Francis Crick in 1958, the Central Dogma outlines the essential processes of DNA replication, transcription, and translation, shaping the way molecular biologists comprehend the intricacies of life. This essay delves into the significance ofthe Central Dogma, elucidating its core components and their crucial role in maintaining and propagating life.

The Central Dogma’s Components:

DNA Replication: DNA (deoxyribonucleic acid) is the hereditary material carrying genetic instructions that define an organism’s traits. Before cell division or reproduction, DNA must be faithfully copied to ensure the accurate transmission of genetic information to the next generation. DNA replication occurs during the cell cycle, involving the unwinding of the double helix and the synthesis of two identical daughter DNA strands, each complementary to one of the original parental strands.

Transcription: The process of transcription occurs in the cell nucleus, where the genetic information encoded in DNA is used to synthesize RNA (ribonucleic acid). RNA serves as a messenger, carrying the genetic code from the nucleus to the cytoplasm, where it participates in protein synthesis. During transcription, an enzyme called RNA polymerase reads the DNA template and synthesizes a complementary RNA strand, resulting in a messenger RNA (mRNA) molecule.

Translation: The final step of the Central Dogma is translation, which takes place in the cell’s cytoplasm. Translation involves converting the genetic code carried by mRNA into a functional protein. This process occurs at the ribosomes, where transfer RNA (tRNA) molecules bring specific amino acids to the ribosome based on the codons (three-nucleotide sequences) on the mRNA. The ribosome links these amino acids together, forming a polypeptide chain that folds into a functional protein.

The Significance of the Central Dogma: The Central Dogma represents a fundamental paradigm in molecular biology and serves as a guiding principle for understanding the molecular basis of life. It highlights the unidirectional flow of genetic information, with DNA acting as the stable repository of genetic instructions that is faithfully copied during cell division. This ensures the genetic continuity across generations, maintaining the genetic identity of organisms.

Additionally, the Central Dogma emphasizes the centrality of proteins as the key players in cellular functions. Proteins, synthesized through translation, carry out essential roles in catalyzing biochemical reactions, providing structural support, regulating gene expression, and facilitating communication within and between cells. Understanding the relationship between DNA, RNA, and proteins is vital for comprehending the molecular basis of diseases, cellular development, and evolutionary processes.

Expanding the Central Dogma: While the Central Dogma provided an initial framework for understanding genetic information, subsequent discoveries have expanded its scope. It has become evident that RNA can also play catalytic roles, acting as enzymes known as ribozymes. Moreover, some viruses can reverse the flow of genetic information through reverse transcription, converting RNA into DNA, challenging the strict unidirectionality originally described by the Central Dogma.

Key Concepts and Mechanisms:

Molecular biology investigates the essential biological processes, including DNA replication, transcription, and translation, that govern cellular activities. The central dogma of molecular biology, proposed by Francis Crick, describes the flow of genetic information from DNA to RNA to protein, with each step regulated by specific enzymes and factors.

The study of gene regulation is another crucial aspect of molecular biology. It involves the investigation of how cells control the expression of genes in response to internal and external signals. Understanding gene regulation has broad implications, as it underpins the development of multicellular organisms, cellular differentiation, and response to environmental cues.

Applications in Biotechnology:

The practical applications of molecular biology in biotechnology have been monumental. Genetic engineering has enabled the production of genetically modified organisms (GMOs) with enhanced traits, leading to increased crop yields, disease resistance, and improved nutritional content.

Additionally, the advent of molecular cloning techniques has facilitated the production of valuable therapeutic proteins, such as insulin and growth hormones, through the use of recombinant DNA technology. This has revolutionized medicine and led to the development of numerous biopharmaceuticals that have saved countless lives.

Medical Impact:

Molecular biology has transformed medical research and diagnosis. Techniques like the polymerase chain reaction (PCR) allow the amplification of specific DNA sequences, aiding in the detection of infectious agents, genetic disorders, and cancer markers. The identification of disease-causing mutations at the molecular level has empowered personalized medicine, enabling tailored treatments for individuals based on their genetic makeup.

Furthermore, advancements in genomics and proteomics have provided a deeper understanding of the genetic basis of diseases, leading to the discovery of potential therapeutic targets and the development of precision medicine approaches.

Conclusion:

Molecular biology has played a pivotal role in unlocking the secrets of life at the cellular level. Through the investigation of DNA, RNA, and proteins, molecular biologists have uncovered the mechanisms that govern biological processes and their regulation. This knowledge has not only broadened our understanding of life but also led to numerous practical applications in biotechnology and medicine. As technology continues to advance, molecular biology remains at the forefront of scientific discovery, promising a future of exciting breakthroughs and transformative impact on our society.

Cell and gene therapy future prospective

What is CCD?

SPR (Surface Plasmon Resonance) technology

Molecular biology

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