Kiran Musunuru
@kiranmusunuru
Followers
3,404
Following
92
Media
220
Statuses
1,191
Cardiologist, geneticist, and gene editor. Author of #TheCRISPRGeneration and #GenomeEditingAPracticalGuide
Philadelphia, USA
Joined March 2009
Don't wanna be here?
Send us removal request.
Explore trending content on Musk Viewer
SEVENTEEN
• 1602148 Tweets
無料クーポン
• 152691 Tweets
Hanni
• 144930 Tweets
Jets
• 121394 Tweets
carat
• 90683 Tweets
連休明け
• 90520 Tweets
Rodgers
• 64750 Tweets
DG X ORM KORNNAPHAT
• 62512 Tweets
अब्दुल कलाम
• 54499 Tweets
पूर्व राष्ट्रपति
• 38152 Tweets
भारत रत्न
• 29742 Tweets
#APJAbdulKalam
• 27504 Tweets
The Next Prince Q9
• 21251 Tweets
महान वैज्ञानिक
• 20398 Tweets
FELIP TAKES ON BBPHSTAGE
• 18148 Tweets
JC BBPH MAINSTAGE
• 17973 Tweets
ミンギュ
• 17121 Tweets
#たっぷりじゃなくてたーーっぷりクワトロ
• 16856 Tweets
うさぎくじ
• 15710 Tweets
金木犀の香り
• 13800 Tweets
バーニス
• 13731 Tweets
मुस्लिम एरिया
• 12878 Tweets
うさぎだらけくじ
• 11250 Tweets
ジョシュア
• 11177 Tweets
スングァン
• 10601 Tweets
エスクプス
• 10581 Tweets
Pinned Tweet
When I started working on
#CRISPR
to fight heart disease >10 years ago, I never imagined it might lead to cures for rare inborn errors of metabolism.
Here’s how it’s happening!
Presented/published today
#ASHG23
#ASHG2023
@GeneticsSociety
plenary +
@AJHGNews
+
@HGGAdvances
🧵
How to use
#CRISPR
gene editing to fight heart disease, the
#1
killer worldwide:
A thread 10+ years in the making.
This one starts in 2009 when I was a postdoc in
@skathire
lab
@MassGeneralNews
/
@broadinstitute
studying ❤ risk factors like cholesterol & triglycerides.
(thread)
21
269
883
2
92
361
A milestone in modern medicine: 1st therapeutic in vivo gene editing in human patients!
From Julian Gillmore,
@intelliatweets
& colleagues out today
@NEJM
:
Here’s an overview of a new potential treatment for transthyretin (ATTR) amyloidosis.
(thread)
18
462
1K
How to use
#CRISPR
gene editing to fight heart disease, the
#1
killer worldwide:
A thread 10+ years in the making.
This one starts in 2009 when I was a postdoc in
@skathire
lab
@MassGeneralNews
/
@broadinstitute
studying ❤ risk factors like cholesterol & triglycerides.
(thread)
21
269
883
The latest chapter in a long-running story, in
@Nature
&
@NatureBiotech
:
To recap, here’s a brief history of in vivo genome editing—focused on 1 gene with lots of 🥇 proofs of concept:
#PCSK9
(apologies to other genes!)
(thread)
6
135
485
The culmination of 10+ years of work to use
#CRISPR
gene editing to fight heart disease!
@ambellinger
@VerveTx
$VERV presenting the interim data from the ongoing heart-1 Phase 1b clinical trial of VERVE-101 base editing for HeFH patients, at
#AHA23
now.
Let’s start!
🧵
How to use
#CRISPR
gene editing to fight heart disease, the
#1
killer worldwide:
A thread 10+ years in the making.
This one starts in 2009 when I was a postdoc in
@skathire
lab
@MassGeneralNews
/
@broadinstitute
studying ❤ risk factors like cholesterol & triglycerides.
(thread)
21
269
883
7
63
327
U.K. approves world’s first CRISPR-based medicine, giving greenlight to therapy for sickle cell, thalassemia via
@statnews
1
59
247
A
#CRISPR
milestone - the first U.S. FDA approval of a gene-editing therapy!
Here’s a quick recap of the story of how it came to be:
“How CRISPR + GWAS Cured Sickle Cell Disease”
🧵
2
66
217
Lulu and Nana, humanity’s first gene-edited babies, both have some CRISPR edits in the CCR5 gene, as He Jiankui (JK) attempted. His goal was to confer HIV resistance.
Unfortunately, the editing turned out quite badly, as shown by *his own data* in *his own manuscript*. (thread)
Today is the 1-year anniversary of
@antonioregalado
breaking the news about Chinese scientist He Jiankui (JK) using gene-edited human embryos to establish pregnancies.
It’s time to break the silence. (thread)
9
106
171
3
136
186
Today is the 1-year anniversary of
@antonioregalado
breaking the news about Chinese scientist He Jiankui (JK) using gene-edited human embryos to establish pregnancies.
It’s time to break the silence. (thread)
9
106
171
19/
Today
@VerveTx
announced new data from non-human primates in which LNP-delivered adenine base editor ⬇️ PCSK9 or ANGPTL3 in the liver.
PCSK9:
67% editing
89%⬇️PCSK9 in blood
59%⬇LDL
ANGPTL3:
60% editing
95%⬇ANGPTL3 in blood
64%⬇TG
19%⬇LDL
2
34
92
It’s a good day for a scientist when—
the protein you worked on for your postdoc is the target of a brand-new FDA-approved drug:
*and*
the protein you worked on for your PhD thesis is featured in the New York Times:
6
9
89
Beautiful metaphor from
@UrnovFyodor
: “Imagine you have a car with a flat tire. Gene therapy is taking a fifth wheel and putting it somewhere on the car and hoping it runs. Gene editing is repairing the flat.”
How Designer DNA Is Changing Medicine
@sciam
2
18
87
If this proves out, a bona fide win for stem cell research!
0
16
77
21/
This is an important step—in a long series of steps—to translate genetic discoveries into therapies to fight the preeminent health threat of the 21st century.
It’s been an enthralling 10 years—let’s see what the next 10 years bring!
from
@ginakolata
11
10
76
In major test for prime editing, scientists successfully correct mutations in monkeys via
@statnews
0
24
72
The pleasure and privilege of working with
@skathire
for the past 13 years has been all mine! Much more to come 🚀
And this guy -
@kiranmusunuru
-
what a spectacular partnership since that first email from him in 2008
asking if he can join my newly minted research lab as a postdoc to learn common disease genetics
Amazing journey.
1
0
56
1
2
71
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema | NEJM
0
17
68
11/
In sum, this is a remarkable demonstration of efficacy and safety of a totally new therapeutic modality in a first-in-human study.
Many congratulations to the team responsible for this groundbreaking accomplishment!
6
13
60
Lipid nanoparticles incorporating a GalNAc ligand enable in vivo liver ANGPTL3 editing in wild-type and somatic LDLR knockout non-human primates
0
7
58
Showing off my pipetting skills on camera...or, more accurately, *onto* the camera...
5
4
59
Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas genome correction in cystic fibrosis models
from
@NatureComms
@DJSiegwart
1
8
58
Charlie was the chair of my thesis committee when I was a graduate student at
@RockefellerUniv
, and one of the nicest guys I’ve ever met—so thrilled to see him win the
#NobelPrize
!
Congratulations to all of the laureates!
BREAKING NEWS:
The 2020
#NobelPrize
in Physiology or Medicine has been awarded jointly to Harvey J. Alter, Michael Houghton and Charles M. Rice “for the discovery of Hepatitis C virus.”
435
16K
32K
2
9
58
Today the American Heart Association
@AHAScience
released a Scientific Statement on genetic testing for inherited cardiovascular diseases, published in
@Circ_Gen
Accompanying commentary by
@CarolynHoMD
#cardiogen
(short thread)
AHA Statement on Genetic Testing for Inherited Cardiovascular Diseases
1
20
34
2
27
47
This Friday, November 29, 2019, is the launch of my book—
The CRISPR Generation: The Story of the World’s First Gene-Edited Babies
eBook at (available now)
Print version at , Amazon, and other sites
#TheCRISPRGeneration
2
28
48
Revolutionary Crispr gene editing speeds from lab to treatment room
1
8
44
A step forward for somatic gene-editing therapy to treat cardiac and skeletal muscle disease
#CRISPR
1
24
44
A wonderful profile of
@skathire
, my long-time mentor, from
@BentheFidler
Heart attacks struck Sek Kathiresan's family. He's devoted his life to stopping them. via
@BioPharmaDive
1
5
40
Just finished this wonderful read from
@KevinADavies
. The definitive history of gene editing right up to the present (just missing the
#NobelPrize
announced the day after it came out). I particularly liked the anecdotes of many key
#CRISPR
contributors.
I heartily recommend it!
*** PUBLICATION DAY *** (Hallelujah!)
A mere 10 years after 'The $1,000 Genome', I'm delighted to publish EDITING HUMANITY: The CRISPR Revolution and the New Era of Genome Editing
@Pegasus_Books
.
I've poured everything into this book. I hope you like it!
14
44
146
2
10
34
Today is Thanksgiving in the U.S.—in the spirit of the holiday, I want to focus on a positive story about CRISPR, instead of the misadventures of He Jiankui:
“How CRISPR + GWAS (May Have) Cured Sickle Cell Disease”
Surely a story about which to be thankful. (thread)
In his manuscript, He Jiankui (JK) barely mentions mosaicism—he didn’t realize (or didn’t care?) that the twins’ embryos were flagrantly mosaic.
But he does say “No off-targets, large deletions or pathogenic cancer gene mutations was observed.”
His data say otherwise. (thread)
1
11
19
1
21
36
16/
If you want tips on reading chromatograms like these, sign up to access a recording of a CRISPR workshop by yours truly at the
@GeneticsSociety
2019 Annual Meeting—membership not required:
1
5
34
17/
Tomorrow, I’ll tackle the other major safety concern with germline editing—off-target mutations.
Spoiler alert: there were off-target mutations in JK’s embryos.
For more details/explanation for today’s thread, refer to
#TheCRISPRGeneration
at
3
6
31
Eli Lilly to pay $200 million for Beam’s share of drugs from gene-editing firm Verve via
@statnews
1
2
34
Nine months in, with no more sickle cell crises. Amazing.
0
4
34
Still reading? You must be interested in genome editing!
If you’re new to editing and want to learn how to do all the types of editing described in this thread, may I recommend my new book:
#GenomeEditingAPracticalGuide
Available here (and elsewhere):
1
6
34
#NobelPrize
for CRISPR!
Congratulations to Jennifer and Emmanuelle!
BREAKING NEWS:
The 2020
#NobelPrize
in Chemistry has been awarded to Emmanuelle Charpentier and Jennifer A. Doudna “for the development of a method for genome editing.”
727
24K
48K
0
4
34
20/
If these results can be reproduced in human patients, they’d rival or surpass the effects of all existing drugs that lower LDL or TG...
...but with a single treatment, rather than pills every day or injections every few weeks/months.
from
@sxbegle
2
8
34
8/
Conclusion: Lulu’s embryo was mosaic—a patchwork of cells with different edits, possibly some with no edits.
Is the same true of Lulu’s body?
I’ll explain tomorrow why mosaicism in the context of human germline editing could have serious, adverse health consequences.
1
8
32
@GeneticsSociety
@AJHGNews
@HGGAdvances
@VerveTx
@ChildrensPhila
@KomorLab
@NicoleGaudelli
@davidrliu
@liugroup
@Domi_niqueB
@mn_whittaker
@Xiao_Wang_Penn
@AhrensNicklas
@PennMedicine
@BKleinstiver
21/
Using a dual AAV vector prime editing system developed by
@JessieRoseDavis
@samagyabanskota
@liugroup
, we delivered the best PEmax/pegRNA/ngRNA combination into R408W PKU mice:
- Phe normalization in 3 weeks (longer than LNPs)
- up to 52% whole-liver corrective editing
1
10
28
UPDATE: new info re: He Jiankui’s (JK’s) targeting of CCR5 in human embryos:
Previously I showed a chromatogram from Lulu’s embryo with 3 alleles (instead of the expected 2)—wild-type, -15bp, and ?
H/t
@biohackinfo
and another anonymous source for ID of the 3rd allele. (thread)
9/
Wondering if the chromatogram even shows the claimed -15bp allele?
With a little patience, you can pick it out of the peaks.
What’s the 3rd allele, then?
I can’t figure it out—as best I can tell, it’s a large insertion of random DNA. If you figure it out, I’d love to know.
2
4
14
3
19
31
2/
Transthyretin (TTR) is expressed in liver, is secreted into blood, and functions as vitamin/hormone transporter.
ATTR amyloidosis is caused by abnormal aggregates of destabilized TTR monomers (normally forms tetramers)—either mutant or wild-type—damaging nerves and/or heart.
1
6
27
6/
There are many positions with 3 peaks. This means there are 3 different alleles in play.
Normal cells have a maximum of 2 alleles at 1 genomic position (chromosome pair).
The best (only?) explanation: different cells in Lulu’s embryo had different CRISPR edits of CCR5.
2
4
26
Gene therapy to regenerate cardiac muscle after a heart attack passes a key test in pigs via
@statnews
0
8
28
The bedeviling problem of pathogenic variants being reclassified as variants of uncertain significance—after it’s too late. (1/2)
via
@WSJ
3
19
26
9/
We wondered: was CRISPR just another gene-editing tool? Would it work as well as ZFNs & TALENs?
Qiurong Ding in my lab compared CRISPR-Cas9 & TALENs head-to-head across many loci in stem cells.
We were stunned: CRISPR-Cas9 blew TALENs out of the water. It wasn’t even close.
1
10
28
6/
In today’s study, LNPs were used to deliver Cas9 mRNA and
#CRISPR
guide RNA into hepatocytes in vivo to disrupt TTR gene via non-homologous end-joining (NHEJ).
In hepatocytes, Cas9 efficiently edited TTR & induced same 1-bp insertion 99% of the time—very consistent outcome.
3
6
24
14/
Out today: a study from
@schwanklab
& colleagues with adenine base editor (ABE) from
@NicoleGaudelli
@davidrliu
@liugroup
, delivered by LNPs into NHPs, to knock down liver PCSK9 by splice site editing, resulting in 32% ⬇️ blood PCSK9 protein + 14% ⬇️ LDL-C levels
1
6
27
CRISPR injected into the blood treats a genetic disease for first time
0
12
25
5/
With exome sequencing so new, 2 students in the lab—
@jpirruccello
&
@DoGenetics
—set up a pipeline from scratch.
On a wintry Saturday afternoon, the pipeline finally spit out the thrilling answer:
Each sibling had 2 nonsense mutations in ANGPTL3—natural gene “knockouts”!
1
4
25
3/
Patisiran, developed by
@Alnylam
, is a lipid nanoparticle (LNP)-delivered siRNA that knocks down TTR mRNA in liver.
In clinical trial, 0.3-mg/kg dose IV every 3 weeks = 81% ⬇️ blood TTR, halting neurologic disease progression with ⬆️ quality of life:
1
5
22
15/
The next big innovation in gene editing was the development of cytosine and adenine base editors by the lab of
@davidrliu
, reported in 2016 & 2017.
These base editors allow for specific C➡️T and A➡️G changes in the genome—more precise, more efficient, and safer than Cas9.
1
6
25
An important warning about giving away your children's DNA data—forever waiving their privacy without their consent.
2
17
23
BREAKING NEWS
The 2023
#NobelPrize
in Physiology or Medicine has been awarded to Katalin Karikó and Drew Weissman for their discoveries concerning nucleoside base modifications that enabled the development of effective mRNA vaccines against COVID-19.
4K
28K
79K
0
1
22
Impressive results for the first patients to receive CRISPR-edited cells to treat beta thalassemia and sickle cell disease (1/2)
4
6
22
@mbeisen
ANGPTL3 was first connected to LDL cholesterol levels by GWAS, ultimately leading to an approved treatment for homozygous familial hypercholesterolemia (evinacumab) and other treatments in development.
0
5
22
10/
The investigators did note the potential for off-target editing at 7 sites in the human genome—observed in 1º human hepatocytes in vitro only at much higher LNP doses than would ever be used in patients—though all are in noncoding DNA (less likely to affect gene function).
1
7
21
16/
Alex Chadwick in my lab
@PennMedicine
used base editing in mouse liver to efficiently introduce nonsense mutations into PCSK9 or ANGPTL3.
With ⬇️ANGPTL3 in mice with high cholesterol, she observed:
>50%⬇️triglycerides
>50%⬇️LDL
A double whammy!
1
5
21
Today is the 3rd anniversary of the announcement of the world’s first gene-edited babies, a scientifically and ethically dubious milestone.
From
@metricausa
, an important update on the health of the CRISPR children—Lulu, Nana, and Amy—in
@NatureBiotech
The CRISPR children. My latest story in
@NatureBiotech
, three years in the making. Thank you so much to those willing to be interviewed. So very many said: Noooo, go away, not taking about that!! Thank you to my editors. My sources. A first 🧵
4
77
267
2
5
20
5/
In a proof-of-concept study published in 2018,
@intelliatweets
demonstrated durable editing of TTR in mouse liver using LNP-delivered CRISPR-Cas9, resulting in >97% ⬇️ blood TTR with single 3-mg/kg LNP dose, stable out to 1 year.
1
4
20
11/
Subsequent work has shown PCSK9 editing persists for months to years in animal models—and might be permanent in human patients.
A one-and-done gene-editing therapy would be a new approach to reducing the 18 million deaths/year from ❤ disease.
1
4
22
Soon after the announcement of a transplanted gene-edited porcine heart into a human patient, now a report of transplanted kidneys!
3
6
22
From burst bubble to medical marvel: How lipid nanoparticles became the future of gene therapy via
@MeganMolteni
@statnews
0
6
21
6/n
That’s a wrap!
One (of many) big implications for the field: the 🚪 is wide open for precise corrective editing to definitively treat grievous genetic diseases centered in the liver, including inborn errors of metabolism.
Stay tuned! 🧬
When I started working on
#CRISPR
to fight heart disease >10 years ago, I never imagined it might lead to cures for rare inborn errors of metabolism.
Here’s how it’s happening!
Presented/published today
#ASHG23
#ASHG2023
@GeneticsSociety
plenary +
@AJHGNews
+
@HGGAdvances
🧵
2
92
361
1
5
21
9/
One patient had 96% ⬇️ blood TTR—essentially complete elimination! (Of note, 2 additional groups of patients receiving higher LNP doses are planned.)
No patient had serious adverse events or significant LFT elevations, with 1 patient having a mild infusion-related reaction.
1
4
19
18/
The challenge now is to translate the gene editing to live human patients.
In 2018,
@skathire
,
@JKeithJoung
, & I were the scientific co-founders of
@VerveTx
, for which I serve as senior scientific advisor and where I’ve been on sabbatical this year.
1
2
21
Jennifer Doudna, Fyodor Urnov want to streamline gene editing studies with Danaher's help
via
@endpts
@leilei_wuu
0
3
20
4/
(This is puzzling. Surely JK understood that his talk at the Second International Summit on Human Genome Editing in Hong Kong would be the most important talk of his career to date. How did he mix up such critical information when making his slide?)
1
3
18
3/
We scoured the literature and found that Dr. Gustav Schonfeld
@WUSTLendo
—an Auschwitz survivor, an immigrant to the US, a foremost expert on lipid metabolism—had identified 4 *healthy* siblings with a “double whammy”:
⬇️⬇️LDL & ⬇️⬇️triglycerides (TG)
1
4
19
10/
Our immediate thoughts: if CRISPR works so well in cells in a dish, would it work in a living animal? Could it be therapy?
When Ding delivered CRISPR-Cas9 targeting PCSK9 into adult mouse liver via an adenoviral vector:
>50% editing
≈90%⬇️PCSK9 in blood
≈40%⬇️cholesterol
2
5
20
7/
While at
@MassGeneralNews
, I met
@JKeithJoung
—a foremost expert on gene editing—and learned from him how to make and use zinc-finger nucleases (ZFNs) to edit cells.
As I was starting my own lab
@HSCRB
, I used ZFNs and then TALENs to edit stem cells for ❤️ disease modeling.
2
4
19
6/
My condemnation was harsh because I understood that JK’s actions were both an ethical fail and a scientific fail.
A single figure from JK’s manuscript is enough to see why I was (and remain) so upset.
If you can’t instantly see what’s wrong—you should NOT gene-edit embryos!
3
7
19
2/
Inspired by work from Boileau/Seidah/Breslow/Horton/Hobbs/Cohen/et al.:
1. Activating PCSK9 mutations = ⬆️LDL cholesterol
2. Inactivating mutations = ⬇️LDL & ⬇️heart disease
3. People with no PCSK9 = ⬇️⬇️LDL & *healthy*!
We asked: are there more PCSK9’s to be discovered?
1
5
18
Edits to a cholesterol gene could stop the biggest killer on earth
3
7
18
In his manuscript, He Jiankui (JK) barely mentions mosaicism—he didn’t realize (or didn’t care?) that the twins’ embryos were flagrantly mosaic.
But he does say “No off-targets, large deletions or pathogenic cancer gene mutations was observed.”
His data say otherwise. (thread)
Lulu and Nana, humanity’s first gene-edited babies, both have some CRISPR edits in the CCR5 gene, as He Jiankui (JK) attempted. His goal was to confer HIV resistance.
Unfortunately, the editing turned out quite badly, as shown by *his own data* in *his own manuscript*. (thread)
3
136
186
1
11
19
5/
If you know what to look for, you can immediately tell there’s something wrong with this picture.
If there are only 2 alleles—one wild-type, one with a deletion—there should be at most 2 peaks in any 1 position of the chromatogram.
Do you see any positions with >2 peaks?
1
3
17
@GeneticsSociety
@AJHGNews
@HGGAdvances
@VerveTx
@ChildrensPhila
@KomorLab
@NicoleGaudelli
@davidrliu
@liugroup
@Domi_niqueB
@mn_whittaker
@Xiao_Wang_Penn
@AhrensNicklas
@PennMedicine
@BKleinstiver
@JessieRoseDavis
@samagyabanskota
22/
Our team has received a $26.5 million grant from
@somaticediting
@NINDSnews
@ncats_nih_gov
to take at least one of these PKU therapies to the clinic, along with therapies for other diseases, in collaboration with
@WPeranteau
@ChildrensPhila
1
3
17
4/
It’s generally understood that JK’s effort was an ethical disaster.
What’s not well known—it was also a scientific disaster.
Though JK’s manuscript hasn’t been published, it reveals just how much of a disaster.
It’s time for the biomedical community to know the full truth.
2
11
17
Researchers cover bases with 2 different gene editing modalities to treat rare disease
via
@FierceBiotech
and Helen Floersh
0
2
18
6/
ANGPTL3 was known to be linked to TG in mice, but not LDL.
That same year, GWAS of 100,000 people by
@skathire
, me, & many colleagues ➡️ common variants near ANGPTL3 linked to TG & LDL.
Thus:
⬇️PCSK9 = safe ⬇️LDL
⬇ANGPTL3 = safe ⬇️LDL & ⬇️TG
= candidate drug targets.
1
5
18
A quantum leap in gene editing technology!
New preprint! How do you insert large genes efficiently + programmatically? CRISPR-based methods are programmable, integrases are efficient. Why not combine them?
PASTE places short (~45bp) integrase landing sites that serve as beacons for insertion (up to 36kb)!
@omarabudayyeh
5
81
405
0
5
16
7/
In non-human primates, there was dose-dependent editing of TTR (up to 73% editing with 6-mg/kg LNP dose, consistent with editing of all hepatocytes) and ⬇️ blood TTR (up to >94% reduction) with single LNP doses, stable out to 1 year.
1
3
16
13/
Xiao Wang in my lab used
@Yecuris
liver-humanized mice—the mouse’s own liver replaced with transplanted human liver cells—to show that Cas9 edits human PCSK9 in human liver cells in vivo efficiently.
Conclusion: if we could get Cas9 into the human liver, it would ⬇️ PCSK9.
1
5
17
8/
Over the coming days, I’m going to tweet at length about the scientific and ethical shortcomings of JK’s actions.
My hope is to bring to light and explain aspects of JK’s actions that have remained unclear, a year later.
Can’t wait? Want the full story in a single read?
1
3
17
13/
There are vocal detractors who bemoan the “billions” in taxpayer money spent on GWAS.
Yes, it was a lot of money.
But even if all that comes from it is a cure for sickle cell disease & beta thalassemia—IMHO that’s not so bad a return on investment.
1
6
17
17/
PCSK9 antibodies were shown to reduce ❤️ disease but have to be injected every few weeks.
It was now clear that gene/base editing of PCSK9 or ANGPTL3 was also a viable approach to reduce ❤️ disease—1-shot therapies with possibly lifelong protection.
1
7
17
2/
Here’s the first piece of evidence.
This is a Sanger sequencing chromatogram of a PCR amplicon of the CCR5 target site, using DNA from cells extracted from Lulu’s embryo prior to pregnancy.
In the manuscript, JK claims there’s a wild-type allele and a 15bp deletion allele.
1
2
16
@GeneticsSociety
@AJHGNews
@HGGAdvances
@VerveTx
@ChildrensPhila
@KomorLab
@NicoleGaudelli
@davidrliu
@liugroup
@Domi_niqueB
@mn_whittaker
@Xiao_Wang_Penn
@AhrensNicklas
@PennMedicine
@BKleinstiver
@JessieRoseDavis
@samagyabanskota
@somaticediting
@NINDSnews
@ncats_nih_gov
@WPeranteau
28/
If we’re successful, here’s our vision for addressing mutational discrimination, for enabling equitable treatment of inborn errors of metabolisms.
Much, much more to come. Stay tuned!
#TranslationalJustice
2
2
16
22/
Here are the non-human primate data presented by
@skathire
on behalf of
@VerveTx
in his keynote talk at
#ISSCR2020
.
2
1
16
5/
There’s a lot of uncertainty when it comes to genetic testing.
But what *is* certain is the indispensable role of genetic counselors in caring for patients both before and after genetic testing.
#GeneticCounselors
@GeneticCouns
2
7
15
15/
We don’t know for sure, because JK’s team never had the chance to evaluate the babies themselves.
Maybe that’s for the best.
1
3
14
12/
That’s it for today. Tomorrow, I’ll start doing a deep dive and analyze some of the key figures from JK’s manuscript.
Again, if you don’t want to wait, you can go to and get the eBook version of
#TheCRISPRGeneration
immediately.
5
3
16
12/
JK showed the chromatograms in his Summit talk. Did he not understand his own data, either before or after the pregnancy?
A savvy viewer could have picked up on the problems (if JK hadn’t rushed through his slides).
@GaetanBurgio
was one of the first to note the mosaicism.
1
2
13
A remarkable advance in gene editing from
@liugroup
and collaborators!
Today we report in
@nature
, in collaboration with the labs of Joseph Mougous and
@VamsiMootha
, the development of a new class of CRISPR-free base editors that enable precision editing of mitochondrial DNA (mtDNA) for the first time. 1/7
28
475
2K
1
2
16