Types of Gene Therapy
defective gene.
Types of Gene Therapy
Somatic-cell therapy
This type of gene therapy is
the only one that has been
performed on humans. Genetic
alterations to somatic cells are
restricted to the person being
treated and cannot be passed on to
his or her offspring.
Germ-line therapy
In germ-line therapy, genetic
alterations are made to germ cells,
such as sperm and eggs, in order
to treat inherited diseases.
Gene Delivery Methods
The most common
technique
is to attach healthy genes to
genetically modified viruses. These
infectious agents, known as
vectors, carry the genes into a
cells nucleus and incorporate them
into the genetic material of the
infected cell.
Chimeraplasty
Another gene-delivery method
still under development ,in which
segments of DNA are inserted into
a cells nucleus. The DNA segment
binds with a defective gene in a
way that helps the cells repair
mechanisms identify and fix the
Original Goal
was to substitute a healthy gene
for a defective one, or to repair a
faulty gene, thereby eliminating
symptoms of disease.
The Most Common Technique
Step 1: Cells are removed from
patient.
Step 2: In the laboratory, a
virus is altered so that it
cannot reproduce.
Step 3: A gene is inserted into
the virus.
Step 4: The altered virus is
mixed with cells from the
patient.
Step 5: The cells from the
patient become genetically
altered.
Step 6: The altered cells are
injected into the patient.
Step 7: The genetically altered
cells produce the desired
protein or hormone.
Some Disorders
(That can be treated by Gene -- using a harmless virus or
vector. The British doctors
Therapy)
are working alongside
Seattle, Washington-based
Cystic Fibrosis
biotech firm Targeted
Hemophilia Genetics Corp., which made
the vector being used in the
Phase I/II trial. It will be
several months before the
success of the procedure
can be properly assessed
Gene Therapy Update but medics said there had
been no complications so
Gene Therapy for far.
Blindness
The move into human
LONDON (Reuters) A team of testing follows 15 years of
laboratory and animal
British doctors has carried out the
experimentation, including
world's first eye operations using
tests on dogs whose vision
gene therapy to try to cure a
was restored to the extent
serious sight disorder, officials said they could navigate a maze
on Tuesday. The group from with ease. Testing it for the
Moorfields Eye Hospital and first time in patients is very
University College London (UCL) important and exciting and
has operated on a small number of represents a huge step
young adults with Leber's towards establishing gene
congenital amaurosis, a type of therapy for the treatment of
inherited childhood blindness many different eye
conditions," Robin Ali,
caused by a single abnormal gene.
professor of human
The condition prevents the molecular genetics at UCL,
retina from detecting light said in a statement. The
properly, resulting in clinical trial was given 1
progressive deterioration million pounds ($2 million)
and severely impaired of funding by Britain's
eyesight. There is no Department of Health, which
effective treatment. The said the pioneering research
new experimental procedure underlined the country's
involves inserting normal leading position in gene
copies of the faulty RPE65 therapy in Europe.
gene into cells of the retina
The idea of using gene
-- the light-sensitive layer of
therapy to fix diseases
cells at the back of the eye
caused by genetic faults has the need to reach multiple
long appealed to scientists, sites in the body. The eye,
although getting the idea to by contrast, is relatively
work in practice has proved straightforward, said
tricky. Some gene therapy Andrew George of London's
approaches have helped Imperial College. The eye is
patients. But one 18-year- good for gene therapy
old volunteer died in a gene because it is a simple organ
therapy experiment in 1999 and it is easy to see what is
and two French boys cured going on. There is hope that
of a rare immune disease once gene therapy is
later developed leukemia. developed in the eye,
Over 70 percent of gene scientists could move on to
therapy trials to date have more complex organs," he
been for cancer, where the said.
process is complicated by