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—..."

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…

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:… 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

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