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.
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
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
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
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
Gene therapy
Introduction
History
Overview
Administration route (ex vivo and in vivo)
Categories (somatic and germline therapy)
Gene delivery methods (physical, chemical and biological)
Viral vectors
Adenovirus vectors
Add not associated virus (AAV) based vectors
Retrovirus vectors
Construction and modification of viral vectors (pseudotyping, serology modification etc. )
Strategies
Gene augmentation therapy
Gene inhibition therapy
Gene targeting,
Assisted killing
Prodrug delivery
Clinical trials on Adenosine deaminase deficiency linked severe combined immunodeficiency syndrome, cystic fibrosis, inherited retinopathies
Recent developments
Gene therapy of cancer
Conclusion
Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemiaD.R. Chandravanshi
Gene therapy is the modern techniques of treatment of various diseases and disorders.
Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases.
It is a technique for correcting defective genes responsible for disease development.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
The first approved gene therapy experiment occurred on September1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
Under the direction of William French Anderson, at the National Institutes of Health (NIH),
INTRODUCTION OF GENE THERAPY, HISTORY OF GENE THERAPY, Process of gene therapy, Methods of gene therapy, Ex vivo gene therapy , In Vivo Gene Therapy , Uses of gene therapy, Target sites for Gene Therapy , Vectors for gene therapy , Viral Vectors, Non Viral Vectors,
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
Gene therapy
Introduction
History
Overview
Administration route (ex vivo and in vivo)
Categories (somatic and germline therapy)
Gene delivery methods (physical, chemical and biological)
Viral vectors
Adenovirus vectors
Add not associated virus (AAV) based vectors
Retrovirus vectors
Construction and modification of viral vectors (pseudotyping, serology modification etc. )
Strategies
Gene augmentation therapy
Gene inhibition therapy
Gene targeting,
Assisted killing
Prodrug delivery
Clinical trials on Adenosine deaminase deficiency linked severe combined immunodeficiency syndrome, cystic fibrosis, inherited retinopathies
Recent developments
Gene therapy of cancer
Conclusion
Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemiaD.R. Chandravanshi
Gene therapy is the modern techniques of treatment of various diseases and disorders.
Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases.
It is a technique for correcting defective genes responsible for disease development.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
The first approved gene therapy experiment occurred on September1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
Under the direction of William French Anderson, at the National Institutes of Health (NIH),
INTRODUCTION OF GENE THERAPY, HISTORY OF GENE THERAPY, Process of gene therapy, Methods of gene therapy, Ex vivo gene therapy , In Vivo Gene Therapy , Uses of gene therapy, Target sites for Gene Therapy , Vectors for gene therapy , Viral Vectors, Non Viral Vectors,
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
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.
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.
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.
Gene therapy involves the insertion of a functioning gene into cells to correct a cellular dysfunction
KEY WORDS : GENETICS, MUTATION , GENETIC ENGINEERING.
Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinson's disease, cancer and even HIV, through a number of different approaches (see video: 'Gene Therapy a new tool to cure human diseases'). A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy, but it has been used with some success.
Dyslipidemia is a medical condition that refers to an abnormal level of blood lipids.
The most common type of dyslipidemia is hyperlipidemia or high lipid levels.
less common form of dyslipidemia: hypolipidemia, abnormally low lipid levels.
Dyslipidemias can affect any lipid parameters including LDL cholesterol levels, HDL cholesterol levels, triglycerides, or a combination of these lipids.
Two categories:
Primary dyslipidemia
Secondary dyslipidemia
Potentiometry is an electrochemical method of Analysis deals with the measurement of electric potential or emf of an electrolyte solution under the condition of constant current.
Potentiometry is the measurement of electrical potential of an electrolyte solution to determine its concentration.
The principle is based on the fact that the potential of the given sample is directly proportional to the concentration of its electro active ions or its activity (pH)
When the pair of electrodes is placed in the sample solution it shows the potential difference by the addition of the titrant or by the change in the concentration of the ions.
The theory of potentiometry is based on the nernst equation.It gives the basic relationship between the potential generated by an electrochemical cell and the concentration of the ions.
The potential E ( Half cell potential) of any electrode is given by nernst equation
Safety pharmacology is a branch of pharmacology with its aim to predict the potential clinical risk profile of new chemical entities (NCEs).
It has the ability to predict the potential off-target drug effects on major organ systems which are associated with exposure in the therapeutic range and above.
As an essential part of the spectrum of drug discovery and development, safety pharmacology studies are generally conducted to determine the relative drug effect on main organs, including respiratory system, central nervous system, and cardiovascular system.Safety pharmacology is an essential part of the drug development process that aims to identify and predict adverse effects prior to clinical trials.
SP studies are described in the international conference on harmonization (ICH) S7A and S7B Guidelines.
A genome is an organism’s complete set of DNA or complete genetic makeup, The entire DNA complement. It describes the identity and the sequence of genes of an organism.
Genomics is the study of entire genomes(structure, function, evolution, mapping, and editing of genomes)
Executing the sequencing and analysis of entire human genome enables more rapid and effective identification of disease associated genes and provide drug companies with pre validated targets.
Proteomics is the systematic high-throughput separation and characterization of proteins within biological systems./ large scale study of protein and their functions.
Proteomics measures protein expression directly, not via gene expression, thus achieving better accuracy. Current work uses 2-dimensional polyacrylamide gel electrophoresis(2D- PAGE) and mass spectrometry.
New separation and characterization technologies, such as protein microarray and high throughput chromatography are being developed.
Prokinetics are the type of drugs which enhances gastrointestinal motility/transit by
increasing the frequency or strength of contractions.
They speed up gastric emptying by enhancing coordinated propulsive motility.
Treat Gastrointestinal symptoms : Abdominal discomfort, Bloating, constipation,
Heart burn, nausea and vomiting. And few gastrointestinal disorders : irritable bowel
Syndrome, gastritis, gastroparesis and functional dyspepsia.
Increases gastric emptying
Relief of gastric stasis
Decreases reflux esophagitis/heart burn
Decreases regurgitation of gastric contents& emesis
Cell signaling / Signal Transduction / Transmembrane signaling.
It is the process by which cells communicate with their environment and respond to external stimuli.
When a signaling molecule(ligand) binds to its receptor, it alters the shape or activity of the receptor, triggering a change inside of the cell such as alteration in the activity of a gene / cell division. Thus the original Intercellular Signal is converted into an Intracellular Signal that triggers as a response.
Introduction to the endocrine system
Growth hormone: Mechanism of Action, secretion, regulation.
Prolactin
Sex hormones
Oral contraceptives
Corticosteroids
A Brief Introduction to Ulcers: What are ulcers, its causes, and symptoms. Classification of Antiulcer drugs and their adverse effects.
List of all the screening models available for Antiulcer drugs.
Few of the models are explained with their Principle, procedures, Evaluation, and assessment.
Flu Vaccine Alert in Bangalore Karnatakaaddon Scans
As flu season approaches, health officials in Bangalore, Karnataka, are urging residents to get their flu vaccinations. The seasonal flu, while common, can lead to severe health complications, particularly for vulnerable populations such as young children, the elderly, and those with underlying health conditions.
Dr. Vidisha Kumari, a leading epidemiologist in Bangalore, emphasizes the importance of getting vaccinated. "The flu vaccine is our best defense against the influenza virus. It not only protects individuals but also helps prevent the spread of the virus in our communities," he says.
This year, the flu season is expected to coincide with a potential increase in other respiratory illnesses. The Karnataka Health Department has launched an awareness campaign highlighting the significance of flu vaccinations. They have set up multiple vaccination centers across Bangalore, making it convenient for residents to receive their shots.
To encourage widespread vaccination, the government is also collaborating with local schools, workplaces, and community centers to facilitate vaccination drives. Special attention is being given to ensuring that the vaccine is accessible to all, including marginalized communities who may have limited access to healthcare.
Residents are reminded that the flu vaccine is safe and effective. Common side effects are mild and may include soreness at the injection site, mild fever, or muscle aches. These side effects are generally short-lived and far less severe than the flu itself.
Healthcare providers are also stressing the importance of continuing COVID-19 precautions. Wearing masks, practicing good hand hygiene, and maintaining social distancing are still crucial, especially in crowded places.
Protect yourself and your loved ones by getting vaccinated. Together, we can help keep Bangalore healthy and safe this flu season. For more information on vaccination centers and schedules, residents can visit the Karnataka Health Department’s official website or follow their social media pages.
Stay informed, stay safe, and get your flu shot today!
Ozempic: Preoperative Management of Patients on GLP-1 Receptor Agonists Saeid Safari
Preoperative Management of Patients on GLP-1 Receptor Agonists like Ozempic and Semiglutide
ASA GUIDELINE
NYSORA Guideline
2 Case Reports of Gastric Ultrasound
MANAGEMENT OF ATRIOVENTRICULAR CONDUCTION BLOCK.pdfJim Jacob Roy
Cardiac conduction defects can occur due to various causes.
Atrioventricular conduction blocks ( AV blocks ) are classified into 3 types.
This document describes the acute management of AV block.
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micro teaching on communication m.sc nursing.pdfAnurag Sharma
Microteaching is a unique model of practice teaching. It is a viable instrument for the. desired change in the teaching behavior or the behavior potential which, in specified types of real. classroom situations, tends to facilitate the achievement of specified types of objectives.
Basavarajeeyam is an important text for ayurvedic physician belonging to andhra pradehs. It is a popular compendium in various parts of our country as well as in andhra pradesh. The content of the text was presented in sanskrit and telugu language (Bilingual). One of the most famous book in ayurvedic pharmaceutics and therapeutics. This book contains 25 chapters called as prakaranas. Many rasaoushadis were explained, pioneer of dhatu druti, nadi pareeksha, mutra pareeksha etc. Belongs to the period of 15-16 century. New diseases like upadamsha, phiranga rogas are explained.
Title: Sense of Taste
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the structure and function of taste buds.
Describe the relationship between the taste threshold and taste index of common substances.
Explain the chemical basis and signal transduction of taste perception for each type of primary taste sensation.
Recognize different abnormalities of taste perception and their causes.
Key Topics:
Significance of Taste Sensation:
Differentiation between pleasant and harmful food
Influence on behavior
Selection of food based on metabolic needs
Receptors of Taste:
Taste buds on the tongue
Influence of sense of smell, texture of food, and pain stimulation (e.g., by pepper)
Primary and Secondary Taste Sensations:
Primary taste sensations: Sweet, Sour, Salty, Bitter, Umami
Chemical basis and signal transduction mechanisms for each taste
Taste Threshold and Index:
Taste threshold values for Sweet (sucrose), Salty (NaCl), Sour (HCl), and Bitter (Quinine)
Taste index relationship: Inversely proportional to taste threshold
Taste Blindness:
Inability to taste certain substances, particularly thiourea compounds
Example: Phenylthiocarbamide
Structure and Function of Taste Buds:
Composition: Epithelial cells, Sustentacular/Supporting cells, Taste cells, Basal cells
Features: Taste pores, Taste hairs/microvilli, and Taste nerve fibers
Location of Taste Buds:
Found in papillae of the tongue (Fungiform, Circumvallate, Foliate)
Also present on the palate, tonsillar pillars, epiglottis, and proximal esophagus
Mechanism of Taste Stimulation:
Interaction of taste substances with receptors on microvilli
Signal transduction pathways for Umami, Sweet, Bitter, Sour, and Salty tastes
Taste Sensitivity and Adaptation:
Decrease in sensitivity with age
Rapid adaptation of taste sensation
Role of Saliva in Taste:
Dissolution of tastants to reach receptors
Washing away the stimulus
Taste Preferences and Aversions:
Mechanisms behind taste preference and aversion
Influence of receptors and neural pathways
Impact of Sensory Nerve Damage:
Degeneration of taste buds if the sensory nerve fiber is cut
Abnormalities of Taste Detection:
Conditions: Ageusia, Hypogeusia, Dysgeusia (parageusia)
Causes: Nerve damage, neurological disorders, infections, poor oral hygiene, adverse drug effects, deficiencies, aging, tobacco use, altered neurotransmitter levels
Neurotransmitters and Taste Threshold:
Effects of serotonin (5-HT) and norepinephrine (NE) on taste sensitivity
Supertasters:
25% of the population with heightened sensitivity to taste, especially bitterness
Increased number of fungiform papillae
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
3. INTRODUCTION
• 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.
3
6. Somatic cell gene therapy Germ cell gene therapy
• Non reproductive cell / Somatic
cells. These are the cells of an
organism other than sperm or
egg cells, e.g., bone marrow
cells, blood cells, skin cells,
intestinal cells.
• somatic cell gene therapy
involves the insertion of a fully
functional and expressible gene
into a target somatic cell to
correct a genetic disease
permanently.
• The genetic alterations in
somatic cells are not carried to
the next generations. Therefore,
somatic cell gene therapy is
preferred .
• The reproductive (sex) cells of
an organism constitute germ
cell line.
• Gene therapy involving the
introduction of DNA into germ
cells is passed on to the
successive generation.
• For safety, ethical and
technical reasons, germ cell
gene therapy is not being
attempted at present.
Approaches in gene therapy
6
7. TYPES OF GENE THERAPY
• Ex vivo gene therapy : This involves the transfer of
genes in cultured cells (ex: bone marrow cells)
which are then reintroduced into the Patient.
• In vivo gene therapy : The direct Transfer of
genes into the cells of a particular tissue is
referred to as in vivo gene therapy.
7
8. EX-VIVO GENE THERAPY
• The ex vivo gene therapy can be applied to only
selected tissues (e.g., bone marrow) whose cells can
be cultured in the laboratory.
• Steps involved
1.lsolate cells with genetic defect from a patient.
2.Grow the cells in culture.
3.Introduce the therapeutic gene to correct gene defect.
4.Select the genetically corrected cells (stable
transformants) and grow.
5.Transplant the modified cells to the patient.
8
9. THE FIRST HUMAN GENE THERAPY
• National Institutes of Health in Bethesda, Maryland, in
1990, Performed on a 4yr old girl Ashanthi DeSilva.
suffering from SCID- Severe Combined Immunodeficiency.
Caused due to defect in gene coding for ADA. Deoxy
adenosine accumulate and destroys T lymphocytes.
Disrupts immunity , suffer from infectious diseases and die
at young age. Correct the deficiency of enzyme, Adenosine
deaminase (ADA).
• Bone marrow cells from the child were transformed with an
engineered retrovirus containing a functional ADA gene.
• The treated cells were reintroduced into the patient’s
marrow. Four years later, the child was leading a normal life
9
10. • synthetic ADA was administered in a complex with
polyethylene glycol (PEG). For many ADA-SCID
patients, injection of the ADA-PEG complex allowed
some immune system development, with weight gain
and reduced infection, although not full immune
reconstitution.
• trial participants received both treatments at once,
making it unclear which treatment was primarily
responsible for the positive clinical outcome.
10
11. IN-VIVO GENE THERAPY
• The direct delivery of the therapeutic gene (DNA) into
the target cells of a particular tissue of a patient
constitutes in vivo gene therapy.
• Many tissues are the potential candidates for this
approach. These include liver, muscle, skin, spleen,
lung, brain and blood cells.
• Gene delivery can be carried out by viral or nonviral
vector systems.
• It can be the only possible option in patients where
individual cells cannot be cultured in vitro in sufficient
numbers (e.g. brain cells).
11
12. • In vivo gene transfer is necessary when cultured cells
cannot be re-implanted in patients effectively.
• The success of in vivo gene therapy mostly depends
on the following parameters.
• The efficiency of the uptake of the remedial
(therapeutic) gene by the target cells.
• lntracellular degradation of the gene and its
uptake by nucleus.
• The expression capability of the gene.
12
13. EXAMPLE:
• In patients with cystic fibrosis, a protein called cystic
fibrosis transmembrane regulator (CFTR) is absent due to
a gene defect.
• In the absence of CFTR chloride ions concentrate within
the cells and it draws water from surrounding.
• This leads to the accumulation of sticky mucous in
respiratory tract and lungs.
• treated by in vivo replacement of defective gene by
adenovirus vector .
13
14. VECTORS IN GENE THERAPY
• The carrier particles or molecules used to deliver
genes to somatic cells are referred to as vectors.
• 2 main classes
• Viral vectors
• Non viral vectors
14
15. VIRAL VECTORS
• The vectors frequently used in gene therapy are viruses,
particularly retroviruses. RNA is the genetic material in
retroviruses. As the retrovirus enters the host cell, it
synthesizes DNA from RNA (by reverse transcription). The
so formed viral DNA (referred to as provirus)gets
incorporated into the DNA of the host cell.
• Risk Factor: some of the retroviruses can convert normal
cells into cancerous ones. Therefore, it is absolutely
essential to ensure that such a thing does not happen.
• Many viral vector systems have been developed for gene
delivery These include retroviruses, adenoviruses, adeno-
associated viruses and herpes simplex virus.
15
16. NON VIRAL VECTORS
• There are certain limitations in using viral vectors in
gene therapy. In addition to the prohibitive cost of
maintaining the viruses, the viral proteins often induce
inflammatory responses in the host.
• Pure DNA constructs that can be directly
introduced into target tissues.
• Lipoplexes, lipid-DNA complexes that have DNA
surrounded by lipid layers.
• Human artificial chromosome which can carry
large DNA (one or more therapeutic genes).
16
17. CLINICAL APPLICATIONS
Bone repair:
Bone loss caused by trauma, neoplasia, reconstructive surgery, congenital
defects or periodontal disease is a major worldwide health problem. The
bone morphogenic proteins (BMPs) enable skeletal tissue formation during
embryogenesis, growth, adulthood, and healing. Probably BMPs (BMPs 2,
4 and 7) are the only growth factors which can singly induce de novo bone
formation both in vitro and at heterotopic sites. Bone defects in the oral and
maxillofacial region can be repaired by transferring genes encoding BMP‘s .
Oral Cancer:
The general strategy in cancer treatment is to express a gene product that
will result in cancer cell death. It can be achieved by Addition of a tumor-
suppressor gene, Deletion of a defective tumor gene, Introduction of genes
to inhibit tumor angiogenesis and "Cancer vaccination" with genes for
tumor antigens.
17
18. • Gene therapy to grow new teeth: This approach is generally
presented in terms of adding molecules to induce de novo tooth
initiation in the mouth. It might be combined with gene-
manipulated tooth regeneration; that is, endogenous dental cells
in situ can be activated or repressed by a gene-delivery technique
to produce a tooth. More than 200 genes are known to be
expressed during tooth development.
• Cardiac disease: Gene therapy has been investigated to target
angiogenesis ( the formation of new blood vessels) during cardiac
surgery and to improve calcium handling mechanism in heart
failure.
• Infectious disease: gene therapy vaccines are being developed
and trailed for tackling infectious diseases including tuberculosis,
malaria, HIV and influenza.
18
19. RECENT ADVANCEMENTS IN GENE THERAPY
2017 Was the Year of Gene-Therapy Breakthroughs
• Sickle-cell cure
In March, researchers announced that a teenage boy in France
had been cured of sickle-cell disease after receiving an
experimental gene therapy developed by Bluebird Bio. Scientists
removed stem cells from the boy’s bone marrow and modified
them in the lab by introducing copies of a gene to prevent his red
blood cells from becoming “sickled.” When the treated cells were
infused back into his body, they began to make normal blood
cells.
• Restoring sight
In December, the FDA approved the first gene therapy for an
inherited disease. The treatment, called Luxturna, aims to correct
a mutation responsible for a range of retinal diseases that make
people gradually go blind. In human tests, the treatment has
restored vision for more than two dozen patients who were losing
their sight.
19
20. • Cancer treatment: The FDA calls the treatment, made by
Novartis, the “first gene therapy” in the U.S. The therapy is
designed to treat an often-lethal type of blood and bone marrow
cancer that affects children and young adults. Known as a CAR-
T therapy, the approach has shown remarkable results in
patients. Kymriah treats a bone marrow cancer that affects
children and young adults, and Yescarta treats a type of
lymphoma.
• Hemophilia : BioMarin is one company working on a gene
therapy that replaces the faulty gene involved in the most
common type of hemophilia, effectively curing the disorder. In
December, the company published early clinical trial
results showing that nine patients who received its therapy saw
substantial increases in the blood-clotting proteins absent in
hemophilia.
20
21. • During 2016, Italian scientists at Milan’s San Raffaele Telethon
Institute for Gene Therapy reported that they had cured 18
children of a rare but terrible immune deficiency disease, ADA-
SCID. They removed the children’s bone marrow, added a gene
to make the ADA enzyme their bodies lack, and replaced
it. Technology Review explained how the treatment, now called
Strimvelis and owned by Glaxo, took 14 years to develop and
test. It was approved in Europe in May of this year.
21
22. REFERENCES
• Biochemistry by U. Satyanarayana , U. Chakrapani 3rd edition
• http://www.rroij.com/open-access/gene-therapy-principles-and-
applications-in-dentistry-5-12.php?aid=34559
• https://journals.sagepub.com/doi/full/10.1177/01926233073099
25
• www.slideshare.com
• https://academic.oup.com/hmg/article/5/Supplement_1/1397/6
61061
22