Genetic disorders can be caused by abnormalities in a single gene or multiple genes, and may be inherited from parents or occur spontaneously. They range from very rare to more common conditions. The inheritance pattern (autosomal dominant, recessive, X-linked, mitochondrial) determines who is at risk of passing on the disorder. While some disorders cause death or disability, others only affect specific traits; treatment focuses on managing symptoms rather than curing the underlying genetic cause.
It is a powerpoint presentation that discusses about the lesson or topic: Sex-Linked Inheritance. It also talks about the definition, and the concepts about Sex-Linked Inheritance.
It is a powerpoint presentation that discusses about the lesson or topic: Sex-Linked Inheritance. It also talks about the definition, and the concepts about Sex-Linked Inheritance.
This presentation is based on genetic disorders. It is a vast topic and I have tried to focus on autosomal disorders along with a general introduction.
Clinical genetics is one of the most rapidly advancing fields in medicine. Spectacular progress has been achieved in this century with unravelling of the entire draft sequence of the human genome. A major contribution of these advances has been in diagnosis, management and prenatal diagnosis of genetic disorders as treatment in most cases is difficult or impossible and where available beyond the means of most families. Genetic technology is advancing rapidly, bringing new, safer and more sensitive ways to diagnose genetic conditions pre- and postnatally. These advances will bring about profound changes in the way we deliver obstetric services to women and their families. Diagnosing a genetic disorder not only allows for disease-specific management options but also has implications for the affected individual's entire family. Hence, a working understanding of the underlying concepts of genetic disease is important for all practicing clinicians. Although it is impossible to know all aspects of clinical and molecular genetics, basic knowledge of certain topics is a must for all practicing obstetrician/gynecologists.
This ppt is prepared by Sandeep Kumar Maurya , m. pharma ,department of pharmaceutical sciences, dr. harisingh gour university sagar madhya pradesh.
This SlideShare covers some of genetic disorders , molecular pathology, single gene disorder type of single gene disorder and advanced level cancer , mechanism of cancer, model for cancer induction explanation.
1. Introduction of genetic disorder
2. Common genetic disorders
3. Causes of genetic disorders
4. Symptoms of genetic disorders
5. single gene disorder
6. Cancer.
8. References.
Genetic disorders occur when a mutation (a harmful change to a gene, also known as a pathogenic variant) affects your genes or when you have the wrong amount of genetic material. Genes are made of DNA (deoxyribonucleic acid), which contain instructions for cell functioning and the characteristics that make you unique.
You receive half your genes from each biological parent and may inherit a gene mutation from one parent or both. Sometimes genes change due to issues within the DNA (mutations). This can raise your risk of having a genetic disorder. Some cause symptoms at birth, while others develop over time.`
Introduction of Cancer
Cancer is caused by the failure of genetic mechanisms that control the growth and proliferation of cells. In most cases, cumulative damage to multiple genes (the "multi-hit" model) via physical and chemical agents, replication errors, etc. contribute to oncogenesis. However, a person's inherited genetic background also may strongly contribute. In cancer, a single transformed cell grows to become a primary tumor, accumulates more mutations and becomes more aggressive, then metastasizes to another tissue and forms a secondary tumor. The difference between a benign tumor and a malignant one mostly involves the latter's ability to invade and metastasize to other tissues. Tumors are classified according to the embryonic origin of the tissue from which they originate. The term carcinoma is used to denote cancers of endodermal (e.g., gut epithelia cancers) or ectodermal (e.g., skin, neural epithelia) origin. Cancers of mesodermal origin (e.g., muscle, blood cells) are called sarcomas. Carcinomas make up >90% of malignant tumors.
This presentation is based on genetic disorders. It is a vast topic and I have tried to focus on autosomal disorders along with a general introduction.
Clinical genetics is one of the most rapidly advancing fields in medicine. Spectacular progress has been achieved in this century with unravelling of the entire draft sequence of the human genome. A major contribution of these advances has been in diagnosis, management and prenatal diagnosis of genetic disorders as treatment in most cases is difficult or impossible and where available beyond the means of most families. Genetic technology is advancing rapidly, bringing new, safer and more sensitive ways to diagnose genetic conditions pre- and postnatally. These advances will bring about profound changes in the way we deliver obstetric services to women and their families. Diagnosing a genetic disorder not only allows for disease-specific management options but also has implications for the affected individual's entire family. Hence, a working understanding of the underlying concepts of genetic disease is important for all practicing clinicians. Although it is impossible to know all aspects of clinical and molecular genetics, basic knowledge of certain topics is a must for all practicing obstetrician/gynecologists.
This ppt is prepared by Sandeep Kumar Maurya , m. pharma ,department of pharmaceutical sciences, dr. harisingh gour university sagar madhya pradesh.
This SlideShare covers some of genetic disorders , molecular pathology, single gene disorder type of single gene disorder and advanced level cancer , mechanism of cancer, model for cancer induction explanation.
1. Introduction of genetic disorder
2. Common genetic disorders
3. Causes of genetic disorders
4. Symptoms of genetic disorders
5. single gene disorder
6. Cancer.
8. References.
Genetic disorders occur when a mutation (a harmful change to a gene, also known as a pathogenic variant) affects your genes or when you have the wrong amount of genetic material. Genes are made of DNA (deoxyribonucleic acid), which contain instructions for cell functioning and the characteristics that make you unique.
You receive half your genes from each biological parent and may inherit a gene mutation from one parent or both. Sometimes genes change due to issues within the DNA (mutations). This can raise your risk of having a genetic disorder. Some cause symptoms at birth, while others develop over time.`
Introduction of Cancer
Cancer is caused by the failure of genetic mechanisms that control the growth and proliferation of cells. In most cases, cumulative damage to multiple genes (the "multi-hit" model) via physical and chemical agents, replication errors, etc. contribute to oncogenesis. However, a person's inherited genetic background also may strongly contribute. In cancer, a single transformed cell grows to become a primary tumor, accumulates more mutations and becomes more aggressive, then metastasizes to another tissue and forms a secondary tumor. The difference between a benign tumor and a malignant one mostly involves the latter's ability to invade and metastasize to other tissues. Tumors are classified according to the embryonic origin of the tissue from which they originate. The term carcinoma is used to denote cancers of endodermal (e.g., gut epithelia cancers) or ectodermal (e.g., skin, neural epithelia) origin. Cancers of mesodermal origin (e.g., muscle, blood cells) are called sarcomas. Carcinomas make up >90% of malignant tumors.
Craniofacial anomalies /certified fixed orthodontic courses by Indian dental ...Indian dental academy
Indian Dental Academy is the Leader in continuing dental education , training dentists in all aspects of dentistry and offering a wide range of dental certified courses in different formats.
The leaflet aims at providing general objective information on genetic tests, including their nature and the potential implications of their results. It presents the different types of tests available, their applications in the medical field and the extent and limit of the significance of the information resulting from these tests.
More information - www.coe.int/bioethics
Cancer cell metabolism: special Reference to Lactate PathwayAADYARAJPANDEY1
Normal Cell Metabolism:
Cellular respiration describes the series of steps that cells use to break down sugar and other chemicals to get the energy we need to function.
Energy is stored in the bonds of glucose and when glucose is broken down, much of that energy is released.
Cell utilize energy in the form of ATP.
The first step of respiration is called glycolysis. In a series of steps, glycolysis breaks glucose into two smaller molecules - a chemical called pyruvate. A small amount of ATP is formed during this process.
Most healthy cells continue the breakdown in a second process, called the Kreb's cycle. The Kreb's cycle allows cells to “burn” the pyruvates made in glycolysis to get more ATP.
The last step in the breakdown of glucose is called oxidative phosphorylation (Ox-Phos).
It takes place in specialized cell structures called mitochondria. This process produces a large amount of ATP. Importantly, cells need oxygen to complete oxidative phosphorylation.
If a cell completes only glycolysis, only 2 molecules of ATP are made per glucose. However, if the cell completes the entire respiration process (glycolysis - Kreb's - oxidative phosphorylation), about 36 molecules of ATP are created, giving it much more energy to use.
IN CANCER CELL:
Unlike healthy cells that "burn" the entire molecule of sugar to capture a large amount of energy as ATP, cancer cells are wasteful.
Cancer cells only partially break down sugar molecules. They overuse the first step of respiration, glycolysis. They frequently do not complete the second step, oxidative phosphorylation.
This results in only 2 molecules of ATP per each glucose molecule instead of the 36 or so ATPs healthy cells gain. As a result, cancer cells need to use a lot more sugar molecules to get enough energy to survive.
Unlike healthy cells that "burn" the entire molecule of sugar to capture a large amount of energy as ATP, cancer cells are wasteful.
Cancer cells only partially break down sugar molecules. They overuse the first step of respiration, glycolysis. They frequently do not complete the second step, oxidative phosphorylation.
This results in only 2 molecules of ATP per each glucose molecule instead of the 36 or so ATPs healthy cells gain. As a result, cancer cells need to use a lot more sugar molecules to get enough energy to survive.
introduction to WARBERG PHENOMENA:
WARBURG EFFECT Usually, cancer cells are highly glycolytic (glucose addiction) and take up more glucose than do normal cells from outside.
Otto Heinrich Warburg (; 8 October 1883 – 1 August 1970) In 1931 was awarded the Nobel Prize in Physiology for his "discovery of the nature and mode of action of the respiratory enzyme.
WARNBURG EFFECT : cancer cells under aerobic (well-oxygenated) conditions to metabolize glucose to lactate (aerobic glycolysis) is known as the Warburg effect. Warburg made the observation that tumor slices consume glucose and secrete lactate at a higher rate than normal tissues.
Earliest Galaxies in the JADES Origins Field: Luminosity Function and Cosmic ...Sérgio Sacani
We characterize the earliest galaxy population in the JADES Origins Field (JOF), the deepest
imaging field observed with JWST. We make use of the ancillary Hubble optical images (5 filters
spanning 0.4−0.9µm) and novel JWST images with 14 filters spanning 0.8−5µm, including 7 mediumband filters, and reaching total exposure times of up to 46 hours per filter. We combine all our data
at > 2.3µm to construct an ultradeep image, reaching as deep as ≈ 31.4 AB mag in the stack and
30.3-31.0 AB mag (5σ, r = 0.1” circular aperture) in individual filters. We measure photometric
redshifts and use robust selection criteria to identify a sample of eight galaxy candidates at redshifts
z = 11.5 − 15. These objects show compact half-light radii of R1/2 ∼ 50 − 200pc, stellar masses of
M⋆ ∼ 107−108M⊙, and star-formation rates of SFR ∼ 0.1−1 M⊙ yr−1
. Our search finds no candidates
at 15 < z < 20, placing upper limits at these redshifts. We develop a forward modeling approach to
infer the properties of the evolving luminosity function without binning in redshift or luminosity that
marginalizes over the photometric redshift uncertainty of our candidate galaxies and incorporates the
impact of non-detections. We find a z = 12 luminosity function in good agreement with prior results,
and that the luminosity function normalization and UV luminosity density decline by a factor of ∼ 2.5
from z = 12 to z = 14. We discuss the possible implications of our results in the context of theoretical
models for evolution of the dark matter halo mass function.
Deep Behavioral Phenotyping in Systems Neuroscience for Functional Atlasing a...Ana Luísa Pinho
Functional Magnetic Resonance Imaging (fMRI) provides means to characterize brain activations in response to behavior. However, cognitive neuroscience has been limited to group-level effects referring to the performance of specific tasks. To obtain the functional profile of elementary cognitive mechanisms, the combination of brain responses to many tasks is required. Yet, to date, both structural atlases and parcellation-based activations do not fully account for cognitive function and still present several limitations. Further, they do not adapt overall to individual characteristics. In this talk, I will give an account of deep-behavioral phenotyping strategies, namely data-driven methods in large task-fMRI datasets, to optimize functional brain-data collection and improve inference of effects-of-interest related to mental processes. Key to this approach is the employment of fast multi-functional paradigms rich on features that can be well parametrized and, consequently, facilitate the creation of psycho-physiological constructs to be modelled with imaging data. Particular emphasis will be given to music stimuli when studying high-order cognitive mechanisms, due to their ecological nature and quality to enable complex behavior compounded by discrete entities. I will also discuss how deep-behavioral phenotyping and individualized models applied to neuroimaging data can better account for the subject-specific organization of domain-general cognitive systems in the human brain. Finally, the accumulation of functional brain signatures brings the possibility to clarify relationships among tasks and create a univocal link between brain systems and mental functions through: (1) the development of ontologies proposing an organization of cognitive processes; and (2) brain-network taxonomies describing functional specialization. To this end, tools to improve commensurability in cognitive science are necessary, such as public repositories, ontology-based platforms and automated meta-analysis tools. I will thus discuss some brain-atlasing resources currently under development, and their applicability in cognitive as well as clinical neuroscience.
Nutraceutical market, scope and growth: Herbal drug technologyLokesh Patil
As consumer awareness of health and wellness rises, the nutraceutical market—which includes goods like functional meals, drinks, and dietary supplements that provide health advantages beyond basic nutrition—is growing significantly. As healthcare expenses rise, the population ages, and people want natural and preventative health solutions more and more, this industry is increasing quickly. Further driving market expansion are product formulation innovations and the use of cutting-edge technology for customized nutrition. With its worldwide reach, the nutraceutical industry is expected to keep growing and provide significant chances for research and investment in a number of categories, including vitamins, minerals, probiotics, and herbal supplements.
Comparing Evolved Extractive Text Summary Scores of Bidirectional Encoder Rep...University of Maribor
Slides from:
11th International Conference on Electrical, Electronics and Computer Engineering (IcETRAN), Niš, 3-6 June 2024
Track: Artificial Intelligence
https://www.etran.rs/2024/en/home-english/
(May 29th, 2024) Advancements in Intravital Microscopy- Insights for Preclini...Scintica Instrumentation
Intravital microscopy (IVM) is a powerful tool utilized to study cellular behavior over time and space in vivo. Much of our understanding of cell biology has been accomplished using various in vitro and ex vivo methods; however, these studies do not necessarily reflect the natural dynamics of biological processes. Unlike traditional cell culture or fixed tissue imaging, IVM allows for the ultra-fast high-resolution imaging of cellular processes over time and space and were studied in its natural environment. Real-time visualization of biological processes in the context of an intact organism helps maintain physiological relevance and provide insights into the progression of disease, response to treatments or developmental processes.
In this webinar we give an overview of advanced applications of the IVM system in preclinical research. IVIM technology is a provider of all-in-one intravital microscopy systems and solutions optimized for in vivo imaging of live animal models at sub-micron resolution. The system’s unique features and user-friendly software enables researchers to probe fast dynamic biological processes such as immune cell tracking, cell-cell interaction as well as vascularization and tumor metastasis with exceptional detail. This webinar will also give an overview of IVM being utilized in drug development, offering a view into the intricate interaction between drugs/nanoparticles and tissues in vivo and allows for the evaluation of therapeutic intervention in a variety of tissues and organs. This interdisciplinary collaboration continues to drive the advancements of novel therapeutic strategies.
Multi-source connectivity as the driver of solar wind variability in the heli...Sérgio Sacani
The ambient solar wind that flls the heliosphere originates from multiple
sources in the solar corona and is highly structured. It is often described
as high-speed, relatively homogeneous, plasma streams from coronal
holes and slow-speed, highly variable, streams whose source regions are
under debate. A key goal of ESA/NASA’s Solar Orbiter mission is to identify
solar wind sources and understand what drives the complexity seen in the
heliosphere. By combining magnetic feld modelling and spectroscopic
techniques with high-resolution observations and measurements, we show
that the solar wind variability detected in situ by Solar Orbiter in March
2022 is driven by spatio-temporal changes in the magnetic connectivity to
multiple sources in the solar atmosphere. The magnetic feld footpoints
connected to the spacecraft moved from the boundaries of a coronal hole
to one active region (12961) and then across to another region (12957). This
is refected in the in situ measurements, which show the transition from fast
to highly Alfvénic then to slow solar wind that is disrupted by the arrival of
a coronal mass ejection. Our results describe solar wind variability at 0.5 au
but are applicable to near-Earth observatories.
What is greenhouse gasses and how many gasses are there to affect the Earth.moosaasad1975
What are greenhouse gasses how they affect the earth and its environment what is the future of the environment and earth how the weather and the climate effects.
Richard's aventures in two entangled wonderlandsRichard Gill
Since the loophole-free Bell experiments of 2020 and the Nobel prizes in physics of 2022, critics of Bell's work have retreated to the fortress of super-determinism. Now, super-determinism is a derogatory word - it just means "determinism". Palmer, Hance and Hossenfelder argue that quantum mechanics and determinism are not incompatible, using a sophisticated mathematical construction based on a subtle thinning of allowed states and measurements in quantum mechanics, such that what is left appears to make Bell's argument fail, without altering the empirical predictions of quantum mechanics. I think however that it is a smoke screen, and the slogan "lost in math" comes to my mind. I will discuss some other recent disproofs of Bell's theorem using the language of causality based on causal graphs. Causal thinking is also central to law and justice. I will mention surprising connections to my work on serial killer nurse cases, in particular the Dutch case of Lucia de Berk and the current UK case of Lucy Letby.
2. A genetic disorder is a genetic problem caused by one or
more abnormalities in the genome, especially a condition that
is present from birth (congenital). Most genetic disorders are
quite rare and affect one person in every several thousands or
millions.
Genetic disorders may or may not be heritable, i.e., passed
down from the parents' genes. In non-heritable genetic
disorders, defects may be caused by new mutations or
changes to the DNA. In such cases, the defect will only be
heritable if it occurs in the germ line. The same disease, such
as some forms of cancer, may be caused by an inherited
genetic condition in some people, by new mutations in other
people, and mainly by environmental causes in still other
people. Whether, when and to what extent a person with the
genetic defect or abnormality will actually suffer from the
disease is almost always affected by the environmental
factors and events in the person's development.
3. A single-gene disorder is the result of a
single mutated gene. Over 4000 human
diseases are caused by single-gene
defects. Single-gene disorders can be
passed on to subsequent generations in
several ways. Genomic imprinting and
uniparental disomy, however, may affect
inheritance patterns. The divisions
between recessive and dominant types
are not "hard and fast", although the
divisions between autosomal and X-linked types are (since the latter
types are distinguished purely based on the chromosomal location of
the gene). For example, achondroplasia is typically considered a
dominant disorder, but children with two genes for achondroplasia
have a severe skeletal disorder of which achondroplasics could be
viewed as carriers. Sickle-cell anemia is also considered a recessive
condition, but heterozygous carriers have increased resistance to
malaria in early childhood, which could be described as a related
dominant condition.
4. Only one mutated copy of the gene
will be necessary for a person to
be affected by an autosomal
dominant disorder. Each affected
person usually has one affected
parent. The chance a child will
inherit the mutated gene is 50%.
Autosomal dominant conditions
sometimes have reduced penetrance, which means although only
one mutated copy is needed, not all individuals who inherit that
mutation go on to develop the disease. Examples of this type of
disorder are Huntington's disease, neurofibromatosis type 1,
neurofibromatosis type 2, Marfan syndrome, hereditary no
polyposis colorectal cancer, hereditary multiple exostoses (a
highly penetrant autosomal dominant disorder),Tuberous
sclerosis, Von Willebrand disease, and acute intermittent
porphyria. Birth defects are also called congenital anomalies.
5. Two copies of the gene must be mutated for
a person to be affected by an autosomal
recessive disorder. An affected person usually
has unaffected parents who each carry a single
copy of the mutated gene (and are referred to
as carriers). Two unaffected people who each
carry one copy of the mutated gene have a
25% risk with each pregnancy of having a
child affected by the disorder. Examples of this
type of disorder are Albinism, Medium-chain
acyl-CoA dehydrogenase deficiency,
cystic fibrosis, sickle-cell disease, Tay-Sachs
disease, Niemann-Pick disease, spinal muscular atrophy, and
Roberts syndrome. Certain other phenotypes, such as wet versus
dry earwax, are also determined in an autosomal recessive
fashion.
6. X-linked dominant disorders are
caused by mutations in genes on
the X chromosome. Only a few
disorders have this inheritance
pattern, with a prime example
being X-linked hypophosphatemia
rickets. Males and females are both
affected in these disorders, with
males typically being more severely
affected than females. Some X-linked
dominant conditions, such as Rett syndrome, incontinentia
pigment type 2, and Aicardi syndrome, are usually fatal in males
either in utero or shortly after birth, and are therefore
predominantly seen in females. Exceptions to this finding are
extremely rare cases in which boys with Klinefelter syndrome
(47,XXY) also inherit an X-linked dominant condition and exhibit
symptoms more similar to those of a female in terms of disease
severity. The chance of passing on an X-linked dominant disorder
differs between men and women.
7. X-linked recessive conditions are
also caused by mutations in genes
on the X chromosome. Males are
more frequently affected than
females, and the chance of passing
on the disorder differs between
men and women. The sons of a
man with an X-linked recessive disorder will not be affected,
and his daughters will carry one copy of the mutated gene. A
woman who is a carrier of an X-linked recessive disorder
(XRXr) has a 50% chance of having sons who are affected and
a 50% chance of having daughters who carry one copy of the
mutated gene and are therefore carriers. X-linked recessive
conditions include the serious diseases hemophilia A,
Duchenne muscular dystrophy, and Lesch-Nyhan syndrome,
as well as common and less serious conditions such as male
pattern baldness and red-green color blindness. X-linked
recessive conditions can sometimes manifest in females due
to skewed X-inactivation or monosomy X (Turner syndrome).
8. V. Y-Linked Disorders
Y-linked disorders, also called
holandric disorders, are caused
by mutations on the Y chromosome.
These conditions display may
only be transmitted from the
heterogametic sex (e.g. male
humans) to offspring of the same
sex. More simply, this means that
Y-linked disorders in humans can
only be passed from men to their
sons; females can never be
affected because they do not possess Y-allosomes.
Y-linked disorders are exceedingly rare but the most well-known
examples typically cause infertility. Reproduction in such
conditions is only possible through the circumvention of
infertility by medical intervention.
9. This type of inheritance,
also known as maternal
inheritance, applies to
genes in mitochondrial
DNA. Because only egg cells
contribute mitochondria to
the developing embryo, only
mothers can pass on
mitochondrial conditions to
their children. An example
of this type of disorder is
Leber's hereditary optic
neuropathy.
10. Genetic disorders may also be complex, multifactorial, or
polygenic, meaning they are likely associated with the effects of
multiple genes in combination with lifestyles and environmental
factors. Multifactorial disorders include heart disease and
diabetes. Although complex disorders often cluster in families,
they do not have a clear-cut pattern of inheritance. This makes it
difficult to determine a person’s risk of inheriting or passing on
these disorders. Complex disorders are also difficult to study and
treat, because the specific factors that cause most of these
disorders have not yet been identified. Studies which aim to
identify the cause of complex disorders can use several
methodological approaches to determine genotype-phenotype
associations. One method, the genotype-first approach, starts by
identifying genetic variants within patients and then determining
the associated clinical manifestations. This is opposed to the more
traditional phenotype-first approach, and may identify causal
factors that have previously been obscured by clinical
heterogeneity, penetrance, and expressivity.
11. Due to the wide range of genetic disorders that are presently
known, diagnosis of a genetic disorder is widely varied and
dependent of the disorder. Most genetic disorders are diagnosed at
birth or during early childhood, however some, such as
Huntington's disease, can escape detection until the patient is well
into adulthood.
The basic aspects of a genetic disorder rests on the inheritance of
genetic material. With an in depth family history, it is possible to
anticipate possible disorders in children which direct medical
professionals to specific tests depending on the disorder and allow
parents the chance to prepare for potential lifestyle changes,
anticipate the possibility of stillbirth, or contemplate termination.
Prenatal diagnosis can detect the presence of characteristic
abnormalities in fatal development through ultrasound, or detect
the presence of characteristic substances via invasive procedures
which involve inserting probes or needles into the uterus such as
in amniocentesis.
12. Not all genetic disorders directly result in death, however there
are no known cures for genetic disorders. Many genetic disorders
affect stages of development such as Down's Syndrome. While
others result in purely physical symptoms such as Muscular
Dystrophy. Other disorders, such as Huntington's Disease show
no signs until adulthood. During the active time of a genetic
disorder, patients mostly rely on maintaining or slowing the
degradation of quality of life and maintain patient autonomy.
This includes physical therapy, pain management, and may
include a selection of alternative medicine programs.
13. The treatment of genetic disorders is an on-going battle with over
1800 gene therapy clinical trials having been completed, are on-
going, or have been approved worldwide.
Despite this, most treatment options revolve around treating the
symptoms of the disorders in an attempt to improve patient quality
of life.
Gene therapy refers to a form of treatment where a healthy gene is
introduced to a patient. This should alleviate the defect caused by
a faulty gene or slow the progression of disease. A major obstacle
has been the delivery of genes to the appropriate cell, tissue, and
organ affected by the disorder. How does one introduce a gene
into the potentially trillions of cells which carry the defective copy?
This question has been the roadblock between understanding the
genetic disorder and correcting the genetic disorder.