The document discusses human genome engineering and the creation of designer babies. It begins by introducing the topic and some key terms like pre-implantation genetic diagnosis and germline engineering. It then covers current methods used like CRISPR/Cas9 gene editing and mitochondria replacement therapy. The document addresses both the benefits of reducing genetic diseases and longer lifespans but also the ethical considerations regarding safety and genetic enhancement. It concludes by discussing some recent successes in the field while noting more research is still needed to fully assess safety.

1. Human Genome Engineering
Noida Institute of Engineering and Technology
(Pharmacy Institute)
Greater Noida
Presented by :-
Vishnu Prabhakar
M.Pharm(Pharmacology)
Submitted to :-
Dr. Saumya Das
Associate Professor
NIET (Pharmacy Institute)
Greater Noida

2. INTRODUCTION
• Human Genome Engineering is now used in various
field’s but recently it become a topic of heat debate when
it is used to produce genetically modified human babies.
• A designer baby is a baby whose genetic makeup has
been selected or altered, often to include a
particular gene or to remove genes associated
with disease.
• This process usually involves analyzing
human embryos to identify genes associated with disease
and selecting embryos which have the desired genetic
makeup - a process known as pre-implantation genetic
diagnosis.
• Delivering the desired genes directly into the cell or
using gene editing technology. This process is known
as germline engineering.
Credit:-5thvoice

3. Human Genome Engineering
• Today, majority of the designer babies are created with an aim to prevent inheriting genetic
defects through the selection of “disease-free” embryos by preimplantation genetic
diagnosis
• Some diseases like cystic fibrosis and b-thalassemia, for instance, could be prevented.
However, when all embryos would carry the “disease genes” from a carrier couple, genetic
modification would be necessary.
• Recently developed gene editing tools allow scientists to create designer babies by active
removal of disease gene from carrier embryos. In United Kingdom (UK), the recent
approval to allow the use of mitochondria replacement therapy has generated a heated
debate. In this presentation, we explain the recent developments in this area and discuss the
ethical aspects

4. METHODS
USED FOR
HUMAN
GENOME
ENGINEERING
• GENOME
EDITING TOOLS
ENGINEERING
NUCLEASES
• Engineering nucleases can
either inactivate or repair a
gene of interest. The major
advantage is that it does not
involve the use of a third
person DNA.
• During embryo editing, the
genes in the germ cells could
also be changed and they will
be passed to the next
generations.
• It is based on CRISPR.
• MITOCHONDRIA
DNA
REPLACEMENT
THERAPY
• The therapy involves the
transfer of the nuclear
genome from the pronuclear
stage zygote of a
mitochondria disease carrier
to an enucleated healthy
donor oocyte.
• The resulting embryo then
carries nuclear DNA from its
parents and presumably
normal mtDNA of the oocyte
donor.
• It is controversial because
the alteration is inheritable.
Credit - mercatornet.com

5. METHODS USED FOR
HUMAN GENOME
ENGINEERING
CRISPR(clustered regularly interspaced short palindromic repeats)
These technologies allow genetic material to be added, removed, or altered at
particular locations in the genome.
The CRISPR-Cas9 system works similarly in the lab. Researchers create a
small piece of RNA with a short "guide" sequence that attaches (binds) to a
specific target sequence of DNA in a genome. The RNA also binds to the
Cas9 enzyme. As in bacteria, the modified RNA is used to recognize the
DNA sequence, and the Cas9 enzyme cuts the DNA at the targeted location.

7. ADVANTAGE OF
CRISPR OVER
OTHER METHOD
• Ability to address any site in the genome
or foreign genome.
• Ability to target multiple DNA site
simultaneously.
• Multifunctional programmability delete,
insert or repair gene.
• Potential one-time curative treatment.
• Simple gene editing tool.
• High potency.

8. BENEFITS OF HUMAN GENOME ENGINEERING
• Longer life: When a baby is genetically modified ,it means that the defective
gene are been taken out. Theoretically these babies like a healthier life with
fewer chances of illness. According to studies, this can add up to 30 year to a
child’s life expectancy.
• No genetic disorders: There are number of illness people catch due to genetic
disorders. These include Alzheimer’s disease, muscular atrophy, down
Syndrome etc. Genetic modification reduces the chances of child being
impacted by many such genetic problems.
• No hereditary disorders: Often, many health conditions such as diabetes,
cancer, obesity, heart ailments are inherited from either of the parent’s families.
Knowing the family history in advance will help genetic scientists to remove
the problematic gene in question from the embryo.

9. ETHICAL CONSIDERATION OF HUMAN
GENOME ENGINEERING
First, the safety of these
procedures has not been
fully established. The
potential for harm is great as
the problems may be passed
on to future generations. [1]
These techniques may be
used for genetic
enhancement and not just
for treatment of genetic
diseases.[1]
In the case of treatment with
mitochondrial transfer, a
healthy oocyte donor is
required, and she may suffer
from the complications and
discomfort of the oocyte
donation procedure such as
ovarian hyperstimulation
syndrome (OHSS).[1]

10. PROBLEMS
ASSOCIATED
WITH HUMAN
GENOME
ENGINEERING

11. FUTURE
POSSIBILITIES
OF HUMAN
GENOME
ENGINEERING
• The primary aim of creating designer babies is to
avoid their having heritable diseases coded by
mutations in DNA.
• We believe that these germline genetic editing
methods should still be considered as experimental
procedures and research should continue to improve
the method and to assess its long term safety.
• Designer babies are babies originated from embryos
created by in-vitro fertilization (IVF) and selected
because of the presence or absence of particular
genes or a baby created by genetic interventions into
pre-implantation embryos in the attempt to influence
the traits the resulting children will have.

12. NEW
SUCESSFUL
DISCOVERIES
IN PAST FEW
YEARS
World’s first baby born with new “3 parent”
technique: The method approved in the UK is called
pronuclear transfer and involves fertilizing both the
mother’s egg and a donor egg with the father’s
sperm.(2016),Mexico
First Same-Sex Couple to have a baby: New
advances in stem cell technology are paving the way
for same-sex couples to have a baby that’s entirely
their own, with their own genes.(Jan 9, 2020).
Designer babies Lulu and Nana: These children are
genetically modified in order to make them resistant
to the HIV virus.(2018).China

13. REFERENCES
• [1]. Ronald T.K. Pang, P.C. Ho, Designer babies, Obstetrics,
Gynecology & Reproductive Medicine, Volume 26, Issue 2, 2016,
Pages 59-60, ISSN 1751-7214.
• https://www.discovermagazine.com/health/the-slow-march-toward-
the-first-same-sex-couple-to-have-a-baby
• https://www.newscientist.com/article/2107219-exclusive-worlds-first-
baby-born-with-new-3-parent-technique/
• https://youtu.be/TdBAHexVYzc

14. Thank you
for listening