Delving into the Latest Updates on CRISPR/Cas9(Jingchu University of Technology) with Synapse

Overview

Basic Info

Drug Type

CRISPR/Cas9

Synonyms-

Target-

Action-

Mechanism-

Therapeutic Areas

NeoplasmsInfectious DiseasesUrogenital Diseases

Active Indication-

Inactive Indication

Human Papillomavirus InfectionHuman Papillomavirus-Related Cervical Intraepithelial NeoplasiaUterine Cervical Cancer+ [1]

Originator Organization

Jingchu University of Technology

Active Organization-

Inactive Organization

Jingchu University of Technology

License Organization-

Drug Highest PhasePendingPhase 1

First Approval Date-

Regulation-

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Efficiency of artificial restriction enzymes toward curing HIV has only been separately examined, using differing delivery vehicles. We compared the in vitro transduction and target-mutagenesis efficiency of consortium plasmid and adenoviral vector delivered HIV-1 pol gene targeting zinc finger nuclease (ZFN) with CRISPR/Cas, Custom-ZFN, CRISPR-Cas-9, and plasmids and vectors (murCTSD_pZFN, pGS-U-gRNA, pCMV-Cas-D01A, Ad5-RGD); cell lines (TZM-bl and ACH-2/J-Lat cells); and the latency reversing agents prostratin, suberoylanilide hydroxamic acid, and phorbol myristate acetate. Cell lines were grown in either Dulbecco's modified Eagle's medium or Roswell Park Memorial Institute with the antibiotics kanamycin, zeocin, and efavirenz. Efficiency was assayed by GFP/luciferase activity and/or validated by yeast MEL1 reporter assay, CEL1 restriction fragment assay, and quantitative reverse transcriptase-polymerase chain reaction (qRT-PCR). Ad5-RGD vectors had better transduction efficiency than murCTSD and pGS-U-gRNA/pCMV-Cas-D01A plasmids. CRISPR/Cas9 exhibited better target-mutagenesis efficiency relative to ZFN (delivered by either plasmid or Ad5 vector) based on gel electrophoresis of pol gene amplicons within ACH-2 and J-Lat cells. Ad-5-RGD vectors enhanced target mutagenesis of ZFN, relative to murCTSD_pZFN plasmids, to levels of CRISPR/Cas9 plasmids. Similar reduction of luciferase activity among TZM-bl treated with Ad5-ZFN vectors relative to CRISPR/Cas-9 and murCTSD_pZFN plasmids was observed on challenge with HIV-1. qRT-PCR of HIV-1 pol gene transcripts affirmed that Ad5 (RGD) vectors enhanced target mutagenesis of ZFN. Whereas CRISPR/Cas-9 may possess inherent superior target-mutagenesis efficiency; the efficiency of ZFN (off-target toxicity withstanding) can be enhanced by altering delivery vehicle from plasmid to Ad5 (RGD) vectors.

Biomaterials ScienceQ2 · ENGINEERING & TECHNOLOGY

Intracellular delivery and biodistribution study of CRISPR/Cas9 ribonucleoprotein loaded bioreducible lipidoid nanoparticles

Q2 · ENGINEERING & TECHNOLOGY

Article

Author: Xu, Qiaobing ; Yang, Liu ; Li, Yamin ; Yu, Yingjie ; Glass, Zachary ; Wang, Ming ; Bolinger, Justin ; Shi, Nicola

A combinatorial library of cationic lipidoids were used as nanocarriers for intracellular delivery of the CRISPR/Cas9 ribonucleoprotein complex.

100 Deals associated with CRISPR/Cas9(Jingchu University of Technology)

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Human Papillomavirus-Related Cervical Intraepithelial Neoplasia	Phase 1	China	Jingchu University of Technology	15 Jan 2018
Human Papillomavirus Infection	Phase 1	China	Jingchu University of Technology	-
Uterine Cervical Cancer	Phase 1	China	Jingchu University of Technology	-
Uterine Cervical Dysplasia	Phase 1	China	Jingchu University of Technology	-

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


CRISPRMED2024	Not Applicable	Hepatolenticular Degeneration	-	CRISPR/Cas9 (H1069Q corrected iPSCs)	jlxvbcurla(itxncuxopp) = cmdoalzitx onwkjyxyix (kfciljmxsn )	-	30 Apr 2024
ASGCT2023	Not Applicable	-	-	CRISPR/Cas9 (R-66S RNP treated)	nuzxuhgnod(rccuvsilts) = RNP-treated SCD HSPCs formed a unique cluster of cells displaying impaired erythroid maturation phenotype with a high abundance of biallelic HBB knockout genotype compared to the untreated sample jlqziorqsj (wwdpzxonjn ) View more	-	01 May 2023
ASGCT2023	Not Applicable	-	-	R-66S RNP+ssODN treated		-	01 May 2023
SSIEM2022	Not Applicable	Phenylketonurias	-	CRISPR/Cas9	zdrbdgygox(plupcoocda) = vvdorqyzyn almplvztil (byjnfevoez ) View more	-	30 Aug 2022
SSIEM2022	Not Applicable	Phenylketonurias	-	CRISPR/Cas9	zdrbdgygox(plupcoocda) = hscuggvicc almplvztil (byjnfevoez ) View more	-	30 Aug 2022
ASGCT2022	Not Applicable	Limb-Girdle Muscular Dystrophy Type 2A CAPN3 mutations	-	CRISPR/Cas9 (CAPN3 c.245CtoT)	hakklbumfs(iaezxiubhc) = qiiylmtvyd apzxgwqjfb (lnysbdrplk )	-	02 May 2022
ASGCT2022	Not Applicable	Limb-Girdle Muscular Dystrophy Type 2A CAPN3 mutations	-	CRISPR/Cas9 (CAPN3 c.1469GtoA)	xtpctiglph(hunvlearnu) = mapnbuxhkd avhnctwtrt (cdjnizoaoa )	-	02 May 2022
ASGCT2022	Not Applicable	-	-	CRISPR-Cas9	okqpfkfmhf(ootsvcroun) = epgxcfaelp ayyszokbxt (xkeccymkea )	-	02 May 2022
ARVO2022	Not Applicable	-	-	CRISPR/Cas9	awxwoyftmo(vhmeiguobq) = wqgstmgufq yopfxiefay (elkwfndvjb )	-	01 May 2022
ASGCT2021	Not Applicable	Muscular Dystrophy, Duchenne	-	CRISPR/Cas9	sesdrgdqbr(orxaxohqky) = wncnlmwgcw xubpykuixv (ofyrwqjvhz )	-	27 Apr 2021
SITC2020	Not Applicable	-	-	CRISPR/Cas9 (Non-virally engineered T cells)	cixovcllce(lxrqgaxzek) = lphfuftidx svimkhkksi (azyajmtumi ) View more	-	09 Nov 2020
SITC2020	Not Applicable	Diffuse Large B-Cell Lymphoma	-	CRISPR Cas9 (Primary T cells)	ibwmnqfksd(rkcslcfaum) = bgiagequxk boxdgimsys (gxiysbkmcp )	-	09 Nov 2020
ASGCT2020	Not Applicable	Hemophilia A	-	CRISPR/Cas9 (Engineered HLECs)	ltloomwkub(qqvwboyguy) = fqqxzyfmte xorhwplaxj (erjqmxjlzs )	-	28 Apr 2020
ASGCT2020	Not Applicable	Hemophilia A	-	CRISPR/Cas9 (Control PLCs)	ltloomwkub(qqvwboyguy) = ohctatnugy xorhwplaxj (erjqmxjlzs )	-	28 Apr 2020