Discover and read the best of Twitter Threads about #Cas9

Most recents (7)

1/As promised & after reading $CRSP latest Q4 2022 #financial #report here is my impression regarding @CRISPRTX latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #BioTech #FinTwit #Genomics #GeneEditing 🧵 CRISPR Therapeutics, a biopharmaceutical company focused on
2/IMO the most significant corporate event in Q4 was $CRSP announcement that regulatory Exa-cel submissions of both #SickleCell & #BetaThalassemia validated in the #EU & #UK & that the @US_FDA BLA submission is on track by the end of Q1 ‘23 - possibly reaching the #markets in ‘23 Regulatory submissions complete for exagamglogene autotemcel
3/Exa-cel/CTX001 - the key program in $CRSP portfolio, is an #autologous Ex-Vivo #CRISPR/#Cas9 #GeneEditing therapy aimed for patients suffering from #TDT or severe #SCD. The latest readout for both programs was phenomenal with 42/44 TDT & 31/31(!) demonstrated remarkable results 3/Exa-cel - formally known as CTX001 and the key program in
Read 17 tweets
1/@GraphiteBio presented preclinical results supporting the use of a single-#cell #RNA #sequencing method to assess #gene correction outcomes in #patients treated with nulabeglogene autogedtemcel (nula-cel) - GPH-101. #BioTech #CRISPR #GeneEditing #Genomics $GRPH #ASH22 GraphiteBio presented precl...
2/GPH101 (nula-cel) is a #CRISPR #Cas9 #GeneEditing autologous #stem #cell-based #therapy in clinical development aimed to treat #SickleCellDisease. GPH101 is designed to directly correct the underlying mutation, thereby decreasing HbS production & restoring HbA expression #ASH22 GPH101 (nula-cel) is a CRIS...
3 $GRPH gene correction platform involves editing hematopoietic #stem cells found in the #bonemarrow that develop into various types of #blood #cells. Since red blood cells lose their genomic DNA during maturation-tracking #GeneEditing in mature cells via sequencing is impossible Graphite bio’s gene correct...
Read 7 tweets
1/As promised & after reading $NTLA latest Q1 2022 financial report here is my analysis regarding @intelliatx latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #GeneEditing #BioTech #FinTwit #Genomics👇
2/NTLA-2001 is the most advanced In-Vivo #CRISPR/#Cas9 #GeneEditing program in $NTLA portfolio & is aimed to treat Transthyretin Amyloidosis - (ATTR). NTLA-2001 could potentially halt & reverse the disease in a “one time” treatment by knocking out the TTR gene with a single dose
3/In February $NTLA has presented an updated clinical data from its Phase 1 study of NTLA-2001 which included 15 patients with ATTR amyloidosis. IMO NTLA-2001 readout looks promising & here is a summary that I wrote shortly after @intelliatx’s presentation of NTLA-2001👇
Read 14 tweets
Critical thread on "Self-Spreading #Vaccines"

Johns Hopkins Bloomberg School of Public Health, Center for #Health Security: Technologies to Address - Global Catastrophic Biological Risks, Oct 9, 2018

#biosurveillance "biological disruption" "bioterrorism events #bioengineering
"As a subset of infectious #disease emergencies, global catastrophic biological risk (#GCBR) is a special category of risk involving #biological #agents—whether naturally emerging or reemerging, deliberately created & released, or #laboratory engineered & escaped—..."

#COVID19
This "could lead to sudden, extraordinary, widespread disaster beyond the collective capability of national & int. orgs & the #privatesector to control."

"Global catastrophic biological risk", "transformational surveillance technologies"

#Foundations #CorporateConsolidation
Read 16 tweets
Intro/1 Lo Stato italiano spende ogni anno in ricerca di
base €6 MLD e in ricerca applicata €3 MLD - in tutto lo
0,5% del PIL - che è la metà di quello che spendono i
Paesi del Nord Europa #UgoAmaldi
Intro/2 Ciò ha conseguenze deleterie sulla nostra
competitività perché la ricerca pubblica è il motore
dell’innovazione tecnologica e dell’introduzione di
nuove forme di lavoro #UgoAmaldi
Intro/3 Il dopo-pandemia è il momento opportuno per
colmare il ritardo italiano nella ricerca pubblica
cominciando con l’aggiungere €1,5 MLD al bilancio 2021
della ricerca pubblica e continuando negli anni
successivi in modo da raggiungere l’1,1% del PIL nel
2026 #UgoAmaldi
Read 57 tweets
Happy to share our latest work on In Vivo Systematic Mutagenesis of a Human Enhancer @AxelVisel @diane_dickel @LenPennacchio @LBNLBioSci @BerkeleyLab

cell.com/cell/fulltext/…

Thread👇 (1/12)
A major challenge in human genomics is how to systematically assess the phenotypic impact of enhancer variants identified in human genetics studies. Mouse transgenics is a powerful tool for this purpose but has limited throughput, high cost, and so-called position effects (2/12)
To overcome these limitations, we developed a high-throughput mouse enhancer-reporter assay (enSERT) that relies on #CRISPR/#Cas9-mediated site-specific integration of a transgene into a safe harbor location of the mouse genome and supports expression in all tissues (3/12)
Read 13 tweets
Hace una semana lanzé un HILO en el que explicaba los orígenes e información básica para entender las herramientas #CRISPR. Gracias a @jm_alarcon ese HILO está recogido en esta web: threadreaderapp.com/thread/9834491… Hoy continuaré con otro en el que hablaré de las aplicaciones. Dentro HILO
Las herramientas #CRISPR nos han cambiado tanto la vida a los investigadores de ciencias de la vida que prácticamente suelo decir que pronto solamente habrá dos tipos de laboratorio: (1) los que YA están usando CRISPR en sus experimentos y (2) los que VAN a usar CRISPR. Y ya está
Lo que está ocurriendo con las #CRISPR y la edición genética de alguna manera recuerda a lo que ocurrió a finales de los años 80, cuando apareció y se instaló la técnica de amplificación del ADN #PCR (reacción de la polimerasa en cadena) que está presente hoy en todas partes.
Read 38 tweets

Related hashtags

Did Thread Reader help you today?

Support us! We are indie developers!


This site is made by just two indie developers on a laptop doing marketing, support and development! Read more about the story.

Become a Premium Member ($3.00/month or $30.00/year) and get exclusive features!

Become Premium

Too expensive? Make a small donation by buying us coffee ($5) or help with server cost ($10)

Donate via Paypal Become our Patreon

Thank you for your support!