Medicine: Expanding possibilities

Gewin, Virginia

doi:10.1038/528s10a

Cited by 2 publications

(2 citation statements)

References 5 publications

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“…SCD is another genetic disorder caused by deficiency of oxygen [43] it affects the red blood cells and form a sickle shape, clogs blood vessels and shows pain full symptoms like such as haemolysis, ischaemia, anaemia and multi-organ injury [44,45]. Several research attempts to correct the mutation by ZFN and TALEN induced HDR with the help of stem cells [46,47].…”

Section: Sickle-cell Diseasementioning

confidence: 99%

Role of CRISPR-Cas/9 in Hematological Disorders: A Review

Sharma¹

2021

ABEB

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According to research crRNA sequence have significance role in targeting DNA [1,2]. To established CRISPR/Cas9 Jinek et al, 2012 introduce crRNA and tracrRNA into a single 100 nucleotide and a guider RNA which helps in the breaking of double stranded DNA and thus successfully genome editing done in human and mouse cells [3]. In the hematopoietic setting, CRISPR/Cas9 gene editing has been applied both in research and in clinical translation studies. CRISPER/Cas9 can be used to treat the clonal hematopoiesis by recapitulate the genetic mutation in patient. The main goal is to restore the immune system by the next generation genomics studies coupled with CRISPR/Cas9.

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Section: Sickle-cell Diseasementioning

confidence: 99%

Role of CRISPR-Cas/9 in Hematological Disorders: A Review

Sharma¹

2021

ABEB

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“…SCD is another human genetic disease that is caused by the single‐nucleotide mutation of the HBB gene. Under deoxygenated conditions, sickle haemoglobin polymerization within erythrocytes deforms red blood cells (RBCs) to assume a sickle shape, clogs blood vessels and results in painful symptoms, such as haemolysis, ischaemia, anaemia and multi‐organ injury (Gewin, ; Hoban et al , ).…”

Section: Applications Of Crispr‐cas9 Technology In Haematological Dismentioning

confidence: 99%

CRISPR‐Cas9 technology and its application in haematological disorders

Zhang

McCarty

2016

Br J Haematol

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Summary The recent advent of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated protein 9 (Cas9) system for precise genome editing has revolutionized methodologies in haematology and oncology studies. CRISPR-Cas9 technology can be used to remove and correct genes or mutations, and to introduce site-specific therapeutic genes in human cells. Inherited haematological disorders represent ideal targets for CRISPR-Cas9-mediated gene therapy. Correcting disease-causing mutations could alleviate disease-related symptoms in the near future. The CRISPR-Cas9 system is also a useful tool for delineating molecular mechanisms involving haematological malignancies. Prior to the use of CRISPR-Cas9-mediated gene correction in humans, appropriate delivery systems with higher efficiency and specificity must be identified, and ethical guidelines for applying the technology with controllable safety must be established. Here, the latest applications of CRISPR-Cas9 technology in haematological disorders, current challenges and future directions are reviewed and discussed.

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Medicine: Expanding possibilities

Cited by 2 publications

References 5 publications

Role of CRISPR-Cas/9 in Hematological Disorders: A Review

Role of CRISPR-Cas/9 in Hematological Disorders: A Review

CRISPR‐Cas9 technology and its application in haematological disorders

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