Gene therapy involves introducing DNA into a patient's cells to treat a disease. There are four main strategies of gene therapy: gene augmentation therapy which adds a functional gene; targeted killing of specific cells like cancer cells; targeted mutation correction; and targeted inhibition of gene expression. Gene therapy shows promise for diseases like cystic fibrosis, hemophilia, cancer and more. Challenges remain around safely delivering genes to the right cells and avoiding immune responses.
Transfection methods (DNA to host cell) Erin Davis
Transfection of DNA to host cell can be done by various methods in lab scale.Gene gun,electroporation,lipofection .These methods are used to transfer DNA to the host cell.
Transfection methods (DNA to host cell) Erin Davis
Transfection of DNA to host cell can be done by various methods in lab scale.Gene gun,electroporation,lipofection .These methods are used to transfer DNA to the host cell.
Gene Therapy, Somatic cell gene therapy, germ line gene therapy, classical gene therapy, non-classical gene therapy, targets of gene therapy, barriers of gene therapy, ex vivo gene therapy, in vivo gene therapy, vectors for gene delivery, antisense therapy
Site directed mutgenesis, OLIGONUCLEOTIDE DIRECTED MUTAGENESIS Vipin Shukla
INTRODUCTION, HISTORY, MUTATION, DIRECTED MUTAGENESIS,BASIC MECHANISM OF SITE DIRECTED MUTAGENESIS,METHOD FOR SITE DIRECTED MUTATIONS,THE SINGLE PRIMER METHOD, CASETTEE MUTAGENESIS, PCR-SITED DIRECTED MUTAGENESIS, APPLICATION OF SITE DIRECTED MUTAGENESIS.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
This presentation focuses on the science of Gene Therapy, the techniques of germ-line and somatic gene therapy and the mechanism of curing diseases and disorders using gene therapy. The presentation starts by discussing some common basic terms from genetics and moves on to the historical development of gene therapy techniques in chronological order. The different types of gene therapy techniques and their mechanisms have been discussed in detail subsequently. In concluding slides, some commercially available gene therapy products are mentioned and challenges of gene-therapy techniques have been highlighted.
Gene Therapy, Somatic cell gene therapy, germ line gene therapy, classical gene therapy, non-classical gene therapy, targets of gene therapy, barriers of gene therapy, ex vivo gene therapy, in vivo gene therapy, vectors for gene delivery, antisense therapy
Site directed mutgenesis, OLIGONUCLEOTIDE DIRECTED MUTAGENESIS Vipin Shukla
INTRODUCTION, HISTORY, MUTATION, DIRECTED MUTAGENESIS,BASIC MECHANISM OF SITE DIRECTED MUTAGENESIS,METHOD FOR SITE DIRECTED MUTATIONS,THE SINGLE PRIMER METHOD, CASETTEE MUTAGENESIS, PCR-SITED DIRECTED MUTAGENESIS, APPLICATION OF SITE DIRECTED MUTAGENESIS.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
This presentation focuses on the science of Gene Therapy, the techniques of germ-line and somatic gene therapy and the mechanism of curing diseases and disorders using gene therapy. The presentation starts by discussing some common basic terms from genetics and moves on to the historical development of gene therapy techniques in chronological order. The different types of gene therapy techniques and their mechanisms have been discussed in detail subsequently. In concluding slides, some commercially available gene therapy products are mentioned and challenges of gene-therapy techniques have been highlighted.
Definition, Gene therapy, types of gene therapy, germline gene therapy, somatic cell gene therapy, basic process of gene therapy and potential targets for gene therapy.
In this slide, You will get to learn abut Gene Therapy and different types of gene therapy. Various method of Gene Therapy and Advantage & Disadvantage and Recent advances in Gene Therapy.
Gene therapy is the process of inserting genes into cells to prevent, treat or cure wide range of diseases. Gene therapy primarily involves genetic manipulations in animals or humans to correct a disease. Gene augmentation therapy: a DNA is inserted into the Genome to replace the missing gene product.Gene inhibition therapy: the antisense gene inhibits the expression of the dominant gene.
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptxPRAMESHPANWAR1
Name of the title: Nucleic Acid-Based Therapeutic Delivery System.
It includes information about nucleic acid, gene therapy, and its type, a method to deliver the desired DNA, i.e., vectors and their types, with proper examples and diagrams, and how these things help in delivering a nucleic acid-based therapeutic drug delivery system.
Epistemic Interaction - tuning interfaces to provide information for AI supportAlan Dix
Paper presented at SYNERGY workshop at AVI 2024, Genoa, Italy. 3rd June 2024
https://alandix.com/academic/papers/synergy2024-epistemic/
As machine learning integrates deeper into human-computer interactions, the concept of epistemic interaction emerges, aiming to refine these interactions to enhance system adaptability. This approach encourages minor, intentional adjustments in user behaviour to enrich the data available for system learning. This paper introduces epistemic interaction within the context of human-system communication, illustrating how deliberate interaction design can improve system understanding and adaptation. Through concrete examples, we demonstrate the potential of epistemic interaction to significantly advance human-computer interaction by leveraging intuitive human communication strategies to inform system design and functionality, offering a novel pathway for enriching user-system engagements.
Neuro-symbolic is not enough, we need neuro-*semantic*Frank van Harmelen
Neuro-symbolic (NeSy) AI is on the rise. However, simply machine learning on just any symbolic structure is not sufficient to really harvest the gains of NeSy. These will only be gained when the symbolic structures have an actual semantics. I give an operational definition of semantics as “predictable inference”.
All of this illustrated with link prediction over knowledge graphs, but the argument is general.
Key Trends Shaping the Future of Infrastructure.pdfCheryl Hung
Keynote at DIGIT West Expo, Glasgow on 29 May 2024.
Cheryl Hung, ochery.com
Sr Director, Infrastructure Ecosystem, Arm.
The key trends across hardware, cloud and open-source; exploring how these areas are likely to mature and develop over the short and long-term, and then considering how organisations can position themselves to adapt and thrive.
LF Energy Webinar: Electrical Grid Modelling and Simulation Through PowSyBl -...DanBrown980551
Do you want to learn how to model and simulate an electrical network from scratch in under an hour?
Then welcome to this PowSyBl workshop, hosted by Rte, the French Transmission System Operator (TSO)!
During the webinar, you will discover the PowSyBl ecosystem as well as handle and study an electrical network through an interactive Python notebook.
PowSyBl is an open source project hosted by LF Energy, which offers a comprehensive set of features for electrical grid modelling and simulation. Among other advanced features, PowSyBl provides:
- A fully editable and extendable library for grid component modelling;
- Visualization tools to display your network;
- Grid simulation tools, such as power flows, security analyses (with or without remedial actions) and sensitivity analyses;
The framework is mostly written in Java, with a Python binding so that Python developers can access PowSyBl functionalities as well.
What you will learn during the webinar:
- For beginners: discover PowSyBl's functionalities through a quick general presentation and the notebook, without needing any expert coding skills;
- For advanced developers: master the skills to efficiently apply PowSyBl functionalities to your real-world scenarios.
JMeter webinar - integration with InfluxDB and GrafanaRTTS
Watch this recorded webinar about real-time monitoring of application performance. See how to integrate Apache JMeter, the open-source leader in performance testing, with InfluxDB, the open-source time-series database, and Grafana, the open-source analytics and visualization application.
In this webinar, we will review the benefits of leveraging InfluxDB and Grafana when executing load tests and demonstrate how these tools are used to visualize performance metrics.
Length: 30 minutes
Session Overview
-------------------------------------------
During this webinar, we will cover the following topics while demonstrating the integrations of JMeter, InfluxDB and Grafana:
- What out-of-the-box solutions are available for real-time monitoring JMeter tests?
- What are the benefits of integrating InfluxDB and Grafana into the load testing stack?
- Which features are provided by Grafana?
- Demonstration of InfluxDB and Grafana using a practice web application
To view the webinar recording, go to:
https://www.rttsweb.com/jmeter-integration-webinar
GraphRAG is All You need? LLM & Knowledge GraphGuy Korland
Guy Korland, CEO and Co-founder of FalkorDB, will review two articles on the integration of language models with knowledge graphs.
1. Unifying Large Language Models and Knowledge Graphs: A Roadmap.
https://arxiv.org/abs/2306.08302
2. Microsoft Research's GraphRAG paper and a review paper on various uses of knowledge graphs:
https://www.microsoft.com/en-us/research/blog/graphrag-unlocking-llm-discovery-on-narrative-private-data/
State of ICS and IoT Cyber Threat Landscape Report 2024 previewPrayukth K V
The IoT and OT threat landscape report has been prepared by the Threat Research Team at Sectrio using data from Sectrio, cyber threat intelligence farming facilities spread across over 85 cities around the world. In addition, Sectrio also runs AI-based advanced threat and payload engagement facilities that serve as sinks to attract and engage sophisticated threat actors, and newer malware including new variants and latent threats that are at an earlier stage of development.
The latest edition of the OT/ICS and IoT security Threat Landscape Report 2024 also covers:
State of global ICS asset and network exposure
Sectoral targets and attacks as well as the cost of ransom
Global APT activity, AI usage, actor and tactic profiles, and implications
Rise in volumes of AI-powered cyberattacks
Major cyber events in 2024
Malware and malicious payload trends
Cyberattack types and targets
Vulnerability exploit attempts on CVEs
Attacks on counties – USA
Expansion of bot farms – how, where, and why
In-depth analysis of the cyber threat landscape across North America, South America, Europe, APAC, and the Middle East
Why are attacks on smart factories rising?
Cyber risk predictions
Axis of attacks – Europe
Systemic attacks in the Middle East
Download the full report from here:
https://sectrio.com/resources/ot-threat-landscape-reports/sectrio-releases-ot-ics-and-iot-security-threat-landscape-report-2024/
Elevating Tactical DDD Patterns Through Object CalisthenicsDorra BARTAGUIZ
After immersing yourself in the blue book and its red counterpart, attending DDD-focused conferences, and applying tactical patterns, you're left with a crucial question: How do I ensure my design is effective? Tactical patterns within Domain-Driven Design (DDD) serve as guiding principles for creating clear and manageable domain models. However, achieving success with these patterns requires additional guidance. Interestingly, we've observed that a set of constraints initially designed for training purposes remarkably aligns with effective pattern implementation, offering a more ‘mechanical’ approach. Let's explore together how Object Calisthenics can elevate the design of your tactical DDD patterns, offering concrete help for those venturing into DDD for the first time!
UiPath Test Automation using UiPath Test Suite series, part 4DianaGray10
Welcome to UiPath Test Automation using UiPath Test Suite series part 4. In this session, we will cover Test Manager overview along with SAP heatmap.
The UiPath Test Manager overview with SAP heatmap webinar offers a concise yet comprehensive exploration of the role of a Test Manager within SAP environments, coupled with the utilization of heatmaps for effective testing strategies.
Participants will gain insights into the responsibilities, challenges, and best practices associated with test management in SAP projects. Additionally, the webinar delves into the significance of heatmaps as a visual aid for identifying testing priorities, areas of risk, and resource allocation within SAP landscapes. Through this session, attendees can expect to enhance their understanding of test management principles while learning practical approaches to optimize testing processes in SAP environments using heatmap visualization techniques
What will you get from this session?
1. Insights into SAP testing best practices
2. Heatmap utilization for testing
3. Optimization of testing processes
4. Demo
Topics covered:
Execution from the test manager
Orchestrator execution result
Defect reporting
SAP heatmap example with demo
Speaker:
Deepak Rai, Automation Practice Lead, Boundaryless Group and UiPath MVP
Securing your Kubernetes cluster_ a step-by-step guide to success !KatiaHIMEUR1
Today, after several years of existence, an extremely active community and an ultra-dynamic ecosystem, Kubernetes has established itself as the de facto standard in container orchestration. Thanks to a wide range of managed services, it has never been so easy to set up a ready-to-use Kubernetes cluster.
However, this ease of use means that the subject of security in Kubernetes is often left for later, or even neglected. This exposes companies to significant risks.
In this talk, I'll show you step-by-step how to secure your Kubernetes cluster for greater peace of mind and reliability.
GDG Cloud Southlake #33: Boule & Rebala: Effective AppSec in SDLC using Deplo...James Anderson
Effective Application Security in Software Delivery lifecycle using Deployment Firewall and DBOM
The modern software delivery process (or the CI/CD process) includes many tools, distributed teams, open-source code, and cloud platforms. Constant focus on speed to release software to market, along with the traditional slow and manual security checks has caused gaps in continuous security as an important piece in the software supply chain. Today organizations feel more susceptible to external and internal cyber threats due to the vast attack surface in their applications supply chain and the lack of end-to-end governance and risk management.
The software team must secure its software delivery process to avoid vulnerability and security breaches. This needs to be achieved with existing tool chains and without extensive rework of the delivery processes. This talk will present strategies and techniques for providing visibility into the true risk of the existing vulnerabilities, preventing the introduction of security issues in the software, resolving vulnerabilities in production environments quickly, and capturing the deployment bill of materials (DBOM).
Speakers:
Bob Boule
Robert Boule is a technology enthusiast with PASSION for technology and making things work along with a knack for helping others understand how things work. He comes with around 20 years of solution engineering experience in application security, software continuous delivery, and SaaS platforms. He is known for his dynamic presentations in CI/CD and application security integrated in software delivery lifecycle.
Gopinath Rebala
Gopinath Rebala is the CTO of OpsMx, where he has overall responsibility for the machine learning and data processing architectures for Secure Software Delivery. Gopi also has a strong connection with our customers, leading design and architecture for strategic implementations. Gopi is a frequent speaker and well-known leader in continuous delivery and integrating security into software delivery.
UiPath Test Automation using UiPath Test Suite series, part 3DianaGray10
Welcome to UiPath Test Automation using UiPath Test Suite series part 3. In this session, we will cover desktop automation along with UI automation.
Topics covered:
UI automation Introduction,
UI automation Sample
Desktop automation flow
Pradeep Chinnala, Senior Consultant Automation Developer @WonderBotz and UiPath MVP
Deepak Rai, Automation Practice Lead, Boundaryless Group and UiPath MVP
Kubernetes & AI - Beauty and the Beast !?! @KCD Istanbul 2024Tobias Schneck
As AI technology is pushing into IT I was wondering myself, as an “infrastructure container kubernetes guy”, how get this fancy AI technology get managed from an infrastructure operational view? Is it possible to apply our lovely cloud native principals as well? What benefit’s both technologies could bring to each other?
Let me take this questions and provide you a short journey through existing deployment models and use cases for AI software. On practical examples, we discuss what cloud/on-premise strategy we may need for applying it to our own infrastructure to get it to work from an enterprise perspective. I want to give an overview about infrastructure requirements and technologies, what could be beneficial or limiting your AI use cases in an enterprise environment. An interactive Demo will give you some insides, what approaches I got already working for real.
2. INTRODUCTION
• Gene therapy is when DNA is introduced into a patient to treat a genetic
disease. The new DNA usually contains a functioning gene to correct the effects
of a disease-causing mutation.
• Gene therapy is a strategy used to treat disease by correcting defective genes or
modifying how genes they are expressed.
• Gene therapy will enable patients to be treated by inserting genes into their
cells rather than administering drugs or subjecting them to surgery.
• A gene that is inserted directly into a cell usually does not function. Instead, a
carrier called a vector is genetically engineered to deliver the gene.
• Gene therapy holds promise for treating a wide range of diseases, such as
cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
3. TYPES OF GENE THERAPY
• Somatic gene therapy: transfer of a section of DNA to any cell of the body that
doesn’t produce sperm or eggs. Effects of gene therapy will not be passed onto the
patient’s children. Somatic cells are nonreproductive, Often the effects of somatic
cell therapy are short-lived. Somatic gene therapy can be broadly split into two
categories:
• Ex vivo: where cells are modified outside the body and then transplanted back in
again. cells from the patient’s blood or bone marrow are removed and grown in the
laboratory.
• In vivo: where genes are changed in cells still in the body.
4. • Germline gene therapy: transfer of a section of DNA to cells that
produce eggs or sperm. Effects of gene therapy will be passed onto the
patient’s children and subsequent generations.
• The therapy alters the genome of future generations to come, the use of
germline therapy due to fears over unknown risks and long-term effects
in future generations is inhibited in various countries.
• In addition, the therapy is very costly.
5. Gene therapy using ADENOVIRUS VECTOR
• A gene that is inserted directly into a cell usually does not
function, instead a vector is used i.e. viruses such as
retroviruses, integrate their genetic material (including the new
gene) into a chromosome in the human cell.
• The vector can be injected or given intravenously (by IV)
directly into a specific tissue in the body, where it is taken up by
individual cells.
• If the treatment is successful, the new gene delivered by the
vector will make a functioning protein.
6.
7. Strategies of gene therapy
• Gene augmentation therapy
• Targeted killing of specific cells
• Targeted mutation correction
• Targeted inhibition of gene expression
8. I. GENE AUGMENTATION THERAPY
• It is used to treat diseases caused by a mutation that stops a
gene from producing a functioning product, such as a protein.
• This therapy adds DNA containing a functional version of the
lost gene back into the cell.
• The new gene produces a functioning product at sufficient
levels to replace the protein that was originally missing.
• This is only successful if the effects of the disease are reversible
or have not resulted in lasting damage to the body.
9. • For example, this can be used to treat loss of function disorders such as
cystic fibrosis by introducing a functional copy of the gene to correct the
disease.
10. II.Targeted killing of specific cells
• Artificial cell killing and immune system assisted cell killing have been popular in the
treatment of cancers.
• The aim is to insert DNA into a diseased cell that causes that cell to die.
• Genes are directed to the target cells and then expressed so as to cause cell killing.
• It can be done by two ways:
1. Direct killing: the inserted DNA contains a “suicide” gene that produces a highly toxic
product which kills the diseased cell.
2. Indirect killing: uses immune-stimulatory genes to provoke or enhance an immune
response against the target cell. The inserted DNA causes expression of a protein that
marks the cells so that the diseased cells are attacked by the body’s natural immune
system.
11. • It is essential with this method that the inserted DNA is targeted
appropriately to avoid the death of cells that are functioning normally
12. III. Targeted mutation correction
• The repair of a genetic defect to restore a functional allele.
• Technical difficulties have meant that it is not sufficiently reliable
to warrant clinical trails.
• In principle, it can be done at different levels: at the gene level
or at the RNA transcript level.
13. IV. TARGETED INHIBITION OF GENE
EXPRESSION
• suitable for treating infectious diseases and some cancers.
• The basis of this therapy is to eliminate the activity of a gene
that encourages the growth of disease-related cells.
• If disease cells display an inappropriate expression of a gene
a variety of different systems can be used specifically to block
the expression of a single gene at the DNA, RNA or Protein
levels.
14. • For example, cancer is sometimes the result of the over-activation of an oncogene. So, by
eliminating the activity of that oncogene through gene inhibition therapy, it is possible to
prevent further cell growth and stop the cancer in its tracks.
15. SUCCESSES
• Successes represent a variety of approaches—different vectors, different target
cell populations, and both in vivo and ex vivo approaches for treating a variety
of disorders.
• Immune deficiencies
• Hereditary blindness
• Hemophilia
• Cancer
• Blood disease
16. Immune deficiencies
• Several inherited immune deficiencies have been treated
successfully with gene therapy.
• Most commonly, blood stem cells are removed from patients, and
retroviruses are used to deliver working copies of the defective
genes.
• Severe Combined Immune Deficiency (SCID) and Adenosine
deaminase (ADA) deficiency.
17. Hereditary blindness
• Gene therapies are being developed to treat several different
types of inherited blindness, especially degenerative forms,
where patients gradually lose the light-sensing cells in their
eyes.
• Most gene-therapy vectors used in the eye are based on AAV
(adeno-associated virus).
• In one small trial of patients with a form of degenerative
blindness called LCA (Leber congenital amaurosis), gene
therapy greatly improved vision for at least a few years.
18. Hemophilia and Cancer
• People with hemophilia are missing proteins that help their blood form
clots.
• In a small trial, researchers successfully used an adeno-associated viral
vector to deliver a gene for Factor IX, the missing clotting protein, to
liver cells. After treatment, most of the patients made at least some
Factor IX, and they had fewer bleeding incidents.
• CANCER: herpes simplex 1 virus was used (which normally causes cold
sores) has been shown to be effective against melanoma that has spread
throughout the body.
19. Blood disease
• Patients with beta-Thalassemia have a defect in the beta-globin gene, which
codes for an oxygen-carrying protein in red blood cells.
• In 2007, a patient received gene therapy for severe beta-Thalassemia. Blood
stem cells were taken from his bone marrow and treated with a retrovirus to
transfer a working copy of the beta-globin gene.
• The modified stem cells were returned to his body, where they gave rise to
healthy red blood cells.
20. Challenges of gene therapy
• Delivering the gene to the right place and switching it on.
• Avoiding the immune response.
• Making sure the new gene doesn’t disrupt the function of other genes.
• cost of gene therapy.
Editor's Notes
Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome
Targeted killing of specific cells. This general approach is popular in cancer gene therapies. Genes are directed to the target cells and then expressed so as to cause cell killing. Direct cell killing is possible if the inserted genes are expressed to produce a lethal toxin (suicide genes), or a gene encoding a prodrug is inserted, conferring susceptibility to killing by a subsequently administered drug. Alternatively, selectively lytic viruses can be used. Indirect cell killing uses immune-stimulatory genes to provoke or enhance an immune response against the target cell.
Because of practical difficulties, this approach has yet to be applied but, in principle, it can be done at different levels: at the gene level (e.g. by gene targeting methods based on homologous recombination); or at the RNA transcript level (e.g. by using particular types of therapeutic ribozymes or therapeutic RNA editing).
Targeted inhibition of gene expression. If disease cells display a novel gene product or inappropriate expression of a gene (as in the case of many cancers, infectious diseases, etc.), a variety of different systems can be used specifically to block the expression of a single gene at the DNA, RNA or protein levels. Allele-specific inhibition of expression may be possible in some cases, permitting therapies for some disorders resulting from dominant negative effects. (The example shows correction of a mutation in a mutant gene by homologous recombination, but mutation correction may also be possible at the RNA level. ODN, oligodeoxynucleotide; TFO, triplex-forming oligonucleotide.)
Severe Combined Immune Deficiency (SCID) was one of the first genetic disorders to be treated successfully with gene therapy, proving that the approach could work. However, the first clinical trials ended when the viral vector triggered leukemia (a type of blood cancer) in some patients. Since then, researchers have begun trials with new, safer viral vectors that are much less likely to cause cancer.
Adenosine deaminase (ADA) deficiency is another inherited immune disorder that has been successfully treated with gene therapy. In multiple small trials, patients' blood stem cells were removed, treated with a retroviral vector to deliver a functional copy of the ADA gene, and then returned to the patients. For the majority of patients in these trials, immune function improved to the point that they no longer needed injections of ADA enzyme. Importantly, none of them developed leukemia.
Encouraging results from animal models (especially mouse, rat, and dog) show that gene therapy has the potential to slow or even reverse vision loss.
The eye turns out to be a convenient compartment for gene therapy. The retina, on the inside of the eye, is both easy to access and partially protected from the immune system. And viruses can't move from the eye to other places in the body. Most gene-therapy vectors used in the eye are based on AAV (adeno-associated virus).
In one small trial of patients with a form of degenerative blindness called LCA (Leber congenital amaurosis), gene therapy greatly improved vision for at least a few years. However, the treatment did not stop the retina from continuing to degenerate. In another trial, 6 out of 9 patients with the degenerative disease choroideremia had improved vision after a virus was used to deliver a functional REP1 gene.
Several promising gene-therapy treatments are under development for cancer. One, a modified version of the herpes simplex 1 virus (which normally causes cold sores) has been shown to be effective against melanoma (a skin cancer) that has spread throughout the body. The treatment, called T-VEC, uses a virus that has been modified so that it will (1) not cause cold sores; (2) kill only cancer cells, not healthy ones; and (3) make signals that attract the patient's own immune cells, helping them learn to recognize and fight cancer cells throughout the body. The virus is injected directly into the patient's tumors. It replicates (makes more of itself) inside the cancer cells until they burst, releasing more viruses that can infect additional cancer cells.
A completely different approach was used in a trial to treat 59 patients with leukemia, a type of blood cancer. The patients' own immune cells were removed and treated with a virus that genetically altered them to recognize a protein that sits on the surface of the cancer cells. After the immune cells were returned to the patients, 26 experienced complete remission.
Avoiding the immune response:
The role of the immune system is to fight off intruders.
Sometimes new genes introduced by gene therapy are considered potentially-harmful intruders.
This can spark an immune response in the patient, that could be harmful to them.
Scientists therefore have the challenge of finding a way to deliver genes without the immune system ‘noticing’.
This is usually by using vectors that are less likely to trigger an immune response
Delivering the gene to the right place and switching it on:
it is crucial that the new gene reaches the right cell
delivering a gene into the wrong cell would be inefficient and could also cause health problems for the patient
even once the right cell has been targeted the gene has to be turned on
cells sometimes obstruct this process by shutting down genes that are showing unusual activity
Many genetic disorders that can be targeted with gene therapy are extremely rare. Gene therapy often requires an individual, case-by-case approach. This may be effective, but may also be very expensive.