Emmanuelle Marie Charpentier

(TÓM TẮT)

PROFILE:
Emmanuelle Marie Charpentier (1968) is a
French professor and researcher in
microbiology, genetics, and biochemistry
Education: Pierre and Marie Curie
University (Bachelor of Science, Master of
Science, Doctor of Philosophy)
Pasteur Institute (Doctor of Philosophy
training)

LÀM VIỆC:
- discovered a regulatory RNA molecule that controls virulence factors in

Streptococcus pyogenes bacteria at University of Vienna

- an assistant research scientist at New York University Medical Center
- a research associate at St. Jude’s Children’s Research Hospital in
Memphis
- Skirball Institute of Biomolecular Medicine in New York
- identified small novel RNAs in the S. pyogenes genome
- recognized with numerous honours and awards
- member of the Royal Swedish Academy of Sciences and the European
Academy of Sciences and Arts
- a Director at the Max Planck Institute for Infection Biology

- co-founded CRISPR Therapeutics

CỘNG TÁC:
- Jörg Vogel at the Max Planck Institute

- Jennifer Anne Doudna

- Elitza Deltcheva, who had been a graduate student in Charpentier’s

laboratory in Vienna
THÀNH TỰU:
- Canada Gairdner International Award (2016)
- Kav li Prize in Nanoscience (2018).

- Royal Swedish Academy of Sciences (2015)

- the European Academy of Sciences and Arts (2018).
- Nobel Laureate in Chemistry on 7th October 2020

She attended the Pierre and Marie Curie University (later part of

Sorbonne University) for undergraduate studies, earning a degree
in biochemistry in 1992. Her graduate studies were carried out at the
Pasteur Institute, where she investigated segments of bacterial DNA that
move around the genome and transfer drug resistance between cells. In
1995 she completed a doctorate in microbiology and remained at the
Pasteur Institute for the next year, working as a postdoctoral researcher.
She continued her postdoctoral studies at Rockefeller University in New
York. After working as an assistant research scientist at New York
University Medical Center, she became a research associate at St. Jude’s
Children’s Research Hospital in Memphis and subsequently the Skirball
Institute of Biomolecular Medicine in New York
In 2002 Charpentier returned to Europe, taking a research position
at the University of Vienna. There she discovered a
regulatory RNA molecule that controls virulence factors
in Streptococcus pyogenes bacteria. With the help of molecular
microbiologist Jörg Vogel at the Max Planck Institute for Infection
Biology in Berlin, Charpentier identified small novel RNAs in the S.
pyogenes genome and started investigating the bacteria’s CRISPR
system, which the organism uses as part of its defense
against viruses. She discovered that the S. pyogenes CRISPR system
consists of three components, tracrRNA (trans-activating CRISPR
RNA), CRISPR RNA, and Cas9 protein—a far simpler organization
than she had anticipated.

In 2009 Charpentier continued her investigation of the CRISPR

system at Umeå Centre for Microbial Research in Sweden. With the
assistance of Elitza Deltcheva, who had been a graduate student in
Charpentier’s laboratory in Vienna, Charpentier showed how the
CRISPR system could cut and modify DNA at specific locations in
the genome. In particular, Deltcheva provided evidence that
tracrRNA and CRISPR RNA interact to guide Cas9 to specific DNA
sequences. Charpentier, Vogel, and Deltcheva reported their
discoveries in 2010. The following year Charpentier met Doudna.
The two researchers quickly set to work on a collaboration that
culminated in their discovery in 2012 of the mechanism by which
Cas9 cleaves DNA. The system subsequently was used with great
success to target and modify specific sequences in the genomes of
various organisms.

In 2013 Charpentier co-founded CRISPR Therapeutics, a company

that employed CRISPR methodology for gene therapy in humans,
with operations in Cambridge, Massachusetts, and headquarters in
Zug, Switzerland. Charpentier was a member of the company’s
scientific advisory board. In 2015, after a two-year stint at Hannover
Medical School in Germany, Charpentier moved her laboratory to
the Max Planck Institute.

Charpentier was recognized with numerous honours and awards,

including the Canada Gairdner International Award (2016) and the
Kavli Prize in Nanoscience (2018). She was an elected member of
the Royal Swedish Academy of Sciences (2015) and the
European Academy of Sciences and Arts (2018).
 Jennifer Anne Doudna
(TÓM TẮT)

PROFILE:
Jennifer Anne Doudna, (1964), American
biochemist; Professor of Biochemistry, Biophysics and
Structural Biology

a degree in chemistry in 1985 from Pomona College;

Harvard University; a Ph.D. in biochemistry; University of Colorado, Yale
University; University of California

- book: A Crack in Creation (2017) “The Code Breaker” (2021)

LÀM VIỆC:

- professor of biochemistry and molecular biology at University of

California

- deduced the three-dimensional structures of RNA molecules

- investigated the control of genetic information

- interested in CRISPR

-organized an effort that called for a moratorium on human genome

editing

CỘNG TÁC:

- Jack W. Szostak (who won the 2009 Nobel Prize for Physiology or
Medicine)

- Thomas Cech at the University of Colorado

- Emmanuelle Marie Charpentier

- Jamie Cate(her husband)

- Kaihong Zhou

THÀNH TỰU:

- a Howard Hughes Medical Institute investigator (from 1997)

- Nobel Laureate in Chemistry on 7th October 2020

- Gruber Prize in Genetics (2015)

- the Canada Gairdner International Award (2016)

- Academy of Achievement’s Golden Plate Award

When asked about her success, Doudna comments that much of it was
down to her luck in having good mentors early on in her career and
having had the freedom to build up her laboratory team with people
with whom she shares a personal chemistry and the same scientific
vision and drive.

https://www.whatisbiotechnology.org/index.php/people/summary/
Doudna (vô link này nếu muốn tìm hiểu thêm)

Doudna first made her name uncovering the basic structure and function
of the first ribozyme, a type of catalytic ribonucleic acid (RNA) that helps
catalyse chemical reactions. This work helped lay the foundation for her
later helping to pioneer CRISPR-Cas 9, a tool that has provided the means
to edit genes on an unprecedented scale and at minimal cost. In addition
to her scientific contributions to CRISPR, Doudna is known for
spearheading the public debate to consider the ethical implications of
using CRISPR-Cas9 to edit human embryos.

Family

Jennifer Anne Doudna was born in Washington DC. She often retreated
into books or exploring the rugged volcanic landscape, beaches and lush
vegetation of the island.(Kahn, Pollack)

Growing up surrounded by the natural beauty and ecological diversity of

Hawaii, Doudna quickly acquired an appreciation for nature and became
fascinated with science. One summer they arranged for her to some
spend time with Don Hermes, a biologist and family friend at the
University of Hawaii. He set her the task with two other students to
investigate how one particular fungus, Phytophthora palmivora, infected
papyrus. During her time with Hermes, Doudna also learnt how to embed
fungus in resin and shave off sections for examining under an electron
microscope. As she says, 'It was my [first] taste of the thrill of scientific
discovery, an experience that I’d read so much about - and it left me
hungering for more.'(Doudna and Sternberg)

Doudna first became interested in biochemistry when she was about 12

or 13. This was inspired by two particular events. The first one was
reading James Watson personal account of his discovery of the double
helix of DNA in his book, which her father left on her bed one rainy
afternoon. The second was when she heard a lecture by a young female
scientist about how normal cells became cancerous.

Doudna shares her interest biochemistry with her husband, Jamie Cate.

Following her doctorate, Doudna spent some time working with Szostak
and then left to take up a postgraduate fellowship in the laboratory of
Thomas Cech at the University of Colorado in Boulder. One of the
attractions of going to work with Cech was that he had just won the
Nobel Prize, in 1989, for discovering the catalytic properties of RNA.
(Marino) He also had the equipment to carry out X-ray diffraction which
would help her decipher the three-dimensional atomic structure of RNA.
With no formal training in x-ray diffraction, Doudna spent many of her
early days in Cech’s laboratory acquiring the skill alongside crystallising
RNA molecules in preparation for imaging them.

In 1994 Doudna left Cech’s laboratory to take up a position as an assistant

professor at Yale University. Six years later she was promoted to become
the Henry Ford II Professor of Molecular Biophysics and Biochemistry. In
2002, Doudna moved to the University of California, Berkeley, where she
was appointed Professor of Biochemistry and Molecular Biology.

Achievements
She helped demonstrate that RNA not merely carries instructions from
DNA for synthesising proteins but also helps catalyse the process.
(Doudna, Szostak) Published in 1989, their work helped revolutionise
RNA research. Seven years later Doudna announced, together with
Cech, the three-dimensional structure of the P4-P6 domain of
the Tetrahymena thermophila group I intron ribozyme, a particular
type of RNA. Working with Cech and others, including Cate - her
future husband, Doudna helped demonstrate that the ribozyme had a
defined shape and an organised structure similar to proteins.(Cate et
al; Marino) By 1998, Doudna and her team had determined the crystal
structure of their first viral RNA - the hepatitis delta virus (HDV), a
human pathogen linked to hepatitis B. By working on the structure of
HDV they hoped to determine how viral RNAs functioned so as to
develop treatments to combat viral disease.

Doudna is now closely linked to the invention of a new tool for gene
editing that has radically reduced the time and work needed to edit the
genome.

A few years later, in March 2011, Doudna went to an American

Society for Microbiology conference in Puerto Rico where she met
Emmanuelle Charpentier, a French microbiologist and geneticist then
based at Umea University in Sweden. Immediately warming to
Charpentier, Doudna agreed to partner with her to find out more and
sent over Martin Jinek, her postdoctoral researcher from the Czech
Republic, to work with her. Thereafter a number of other researchers
came on board, including Michael Hauser, a master’s student from
Germany who worked in Doudna Laboratory, and Krystztof Chylinski
a Polish doctorate student of Charpentier who was based in her old
laboratory at the University of Vienna.(Doudna and Sternberg)

Doudna has won numerous awards in her time and on 7th October
2020 she was jointly awarded Nobel Prize for Chemistry together with
Emanuelle Charpentier for their development of gene editing. When
asked about her success, Doudna comments that much of it was
down to her luck in having good mentors early on in her career and
having had the freedom to build up her laboratory team with people
with whom she shares a personal chemistry and the same scientific
vision and drive. A key essence for her is to have laboratory with a
supportive environment where people work together as a team and
older members are prepared to mentor those who are younger. Much
of her achievement she also attributes to Kaihong Zhou, her
laboratory manager, who has worked with her for over thirty years,
starting from when Doudna was at Yale University. The two of them
confer on everything all the way from what projects to run through to
what staff to hire and how to allocate funds. (Mukhopadyay)

Doudna spent much of her youth in Hilo, Hawaii. After earning a

degree in chemistry in 1985 from Pomona College in California, she
went to Harvard University. There she worked in the laboratory of
English-born American biochemist and geneticist Jack W.
Szostak (who won the 2009 Nobel Prize for Physiology or Medicine)
and in 1989 completed a Ph.D. in biochemistry. In 1994, following
postdoctoral studies at the University of Colorado under the
direction of American biochemist and molecular biologist Thomas
R. Cech (who received a share of the 1989 Nobel Prize for
Chemistry), she joined the faculty at Yale University. In 2002 she
moved to the University of California, Berkeley, where she served as
professor of biochemistry and molecular biology.

Early in her career Doudna worked to deduce the three-dimensional

structures of RNA molecules, which provided insight on RNA
catalytic activity. She later investigated the control of genetic
information by certain small RNAs and became interested in
CRISPR. CRISPR is part of the bacterial immune system. It
originates with RNA sequences from invading viruses that become
incorporated into bacterial genomes. The viral sequences reside as
DNA in the spacers between short repeating blocks of bacterial DNA
sequences. The next time the virus invades the bacterial cell, the
spacer DNA is converted to RNA. The Cas9 enzyme and a second
RNA molecule attach to the newly coded RNA, which then seeks out
matching strands of viral DNA. When encountered, Cas9 cuts the
viral DNA, preventing the virus’s replication. Doudna and
Charpentier found that the guide RNA sequence could be changed
to direct Cas9 to a precise DNA sequence. Their discovery quickly
transformed the landscape of genome engineering, creating new
opportunities for the treatment of human disease.

Genome engineering in humans was an inevitable result of rapid

advances in genetic engineering technologies. However, little was
known about its safety, and its use to edit human DNA
renewed ethical concerns, particularly about whether genetic
engineering technologies should be used to modify nondisease
traits, such as intelligence. In early 2015 Doudna organized an effort
that called for a moratorium on human genome editing, and in April
of that year she and colleagues laid out a framework for immediate
actions to safeguard the genomes of human embryos against
modification. Despite the precautionary effort, however, in April
2015 Chinese scientists reported having altered human embryo
genomes via CRISPR-Cas9.

In addition to receiving the Nobel Prize, Doudna received numerous

honours and awards for her research, including the Gruber Prize in
Genetics (2015) and the Canada Gairdner International Award
(2016), both shared with Charpentier. Doudna was an elected
member of multiple academies and a Howard Hughes Medical
Institute investigator (from 1997).