TABLE 1
| Disease | Target gene | Model (animal or cell) | Findings | References |
| β-thalassemia | HBB | CD34+ HSPCs of β-thalassemia patients | ▪ 93.0% indel frequency (SpCas9) | Xu et al. (2021) |
| β-thalassemia | KLF1 | human myeloid leukemia (K562) cell line | ▪ The average indel percentage in the ce ... | Shariati et al. (2016) |
| KLF1 | human myeloid leukemia (K562) cell line | ▪ Relative quantification was performed ... | Shariati et al. (2016) | |
| KLF1 | human myeloid leukemia (K562) cell line | ▪ The levels of γ-globin mRNA on day 5 o ... | Shariati et al. (2016) |
What are the clinical applications of CRISPR/Cas9?
7 rows · Aug 30, 2016 · In addition to genetic diseases, CRISPR/Cas9 gene editing has also been applied in ...
What diseases can be treated with CRISPR?
Mar 21, 2022 · Another potential clinical application of CRISPR/Cas9 is to treat infectious diseases, such as HIV. Although antiretroviral therapy provides an effective treatment for HIV, no cure currently exists due to permanent integration of the virus into the host genome. Hu et al showed the CRISPR/Cas9 system could be used to target HIV-1 genome activity. This …
Can CRISPR-Cas9 be used to treat Alzheimer’s disease?
Jan 25, 2022 · We summarize the potential target genes for immunomodulation using CRISPR-Cas9 in autoimmune diseases including rheumatoid arthritis (RA), inflammatory bowel diseases (IBD), systemic lupus erythematosus (SLE), multiple sclerosis (MS), type 1 diabetes mellitus (DM), psoriasis, and type 1 coeliac disease.
Can CRISPR/Cas9 treat Huntington's disease?
The implementation of the CRISPR-Cas9 system has increased the number of available technological alternatives for studying gene function, thus enabling generation of CRISPR-based disease models. Although many mechanistic questions remain to be answered and several challenges have yet to be addressed, the use of CRISPR-Cas9-based genome ...
What diseases can CRISPR treat?
Eight Diseases CRISPR Technology Could CureCancer. China has been spearheading the first clinical trials using CRISPR-Cas9 as a cancer treatment. ... Blood disorders. ... Blindness. ... AIDS. ... Cystic fibrosis. ... Muscular dystrophy. ... Huntington's disease. ... Covid-19.Sep 13, 2021
What has CRISPR-Cas9 been used for?
The company used it to improve the immunity of bacterial cultures against viruses and many food manufacturers now use the technology to produce cheese and yoghurt. Since then the technology has been used to delete, insert and modify DNA in human cells and other animal cells grown in petri dishes.
How could CRISPR help treat genetic diseases like DMD or hemophilia?
CRISPR-Cas can be used to permanently repair the mutated DMD gene, leading to the expression of the encoded protein, dystrophin, in systems ranging from cells derived from DMD patients to animal models of DMD.Apr 3, 2021
When was CRISPR-Cas9 first used?
The use of CRISPR-Cas9 to edit genes was thrust into the spotlight in 2012 when George Church, Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang harnessed it as a tool to modify targeted regions of genomes. Given its potential to revolutionize gene editing, Science named CRISPR Breakthrough of the Year in 2015.Jun 30, 2020
What is the CRISPR-CAS9 system?
The CRISPR-Cas9 system was discovered from the adaptive immune system of prokaryotes, basically from that of bacteria and archaea [23]. Type II CRISPR-Cas9 is a commonly used system that consists of three core components: the endonuclease Cas9, CRISPR RNA (crRNA), and trans-activating crRNA (tracrRNA) [24].
What is the most commonly used viral vector?
The most commonly used viral vector is AAV owing to its mild immunogenicity, high infection ability, and inefficient to integrate into the human genome generally [45, 46]. The AAV genome consists of a single-stranded DNA, with greater than 200 variants [47].
What is the cause of EOAD?
Genetically most EOAD is caused by dominantly inherited mutations in amyloid-β precursor protein (APP), presenilin-1 (PSEN1), and presenilin-2 (PSEN2) genes. Globally more than 400 mutations were reported inAPP,PSEN1, andPSEN2genes that result in change of Aβ production level (alzforum.org/mutations) [11].
What is the sgRNA sequence?
The sgRNA consists of a 20-nucleotide guide sequence that is complementary to the target site. When the sgRNA recognizes the target sequence, it binds by Watson–Crick base-pairing and guides Cas9 to cleave the DNA strand and forms a double-stranded break (DSB) at the target site.
What are the risk factors for late onset AD?
The major risk factors for late-onset AD are the apolipoprotein E4 (APOE4) allele and mutation in theAPOEgene that transcribes apolipoprotein E protein [36, 37]. Human APOE is polymorphic with three major isoforms, APOE2, APOE3, and APOE4 [38].
What is the rarest form of APOE?
The scarcest form of APOE is E2 while carrying one copy seems to reduce the risk of developing AD by up to 40%. APOE3 is the normal form and does not appear as a risk factor but APOE4 exists in nearly 10–15% of people, increasing the risk for AD.
How many people are affected by AD?
The number of patients with AD is alarmingly increasing worldwide; currently, at least 50 million people are thought to be living with AD. The mutations or alterations in amyloid-β precursor protein (APP), presenilin-1 (PSEN1), or presenilin-2 (PSEN2) genes are known to be associated with the pathophysiology of AD.