Category Archives: CRISPR

Gene editing target disease ?

Genome Editing with CRISPR-Cas9

Here’s a short list of some common diseases that might be curable or preventable with gene editing:

Breast cancer
Colon cancer
Huntington’s disease
Marfan, Parkinson’s
Prostate cancer
Retinitis pigmentosa
Sickle cell
Skin cancer
Duchenne muscular dystrophy
Color blindness
Cystic fibrosis
Down syndrome
Polycystic kidney
Turner syndrome.

There are hundreds of other more rare genetic disorders.… Read more


A new genetic engineering technology called CRISPR/Cas9 has opened the door to unprecedented tinkering with the human genome. Nature takes a look at the scientific possibilities and the ethical dilemmas they raise.… Read more

Gene Therapy and regenerative medicine companies.



Tim Miller, Ph.D., President & CEO
Dallas, TX
PlasmaTech Biopharmaceuticals/Abeona Therapeutics is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. PlasmaTech’s lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary

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How CRISPR-Cas9 Works ?

Researchers are developing ways to use CRISPR to treat genetic conditions like sickle-cell anemia and cystic fibrosis, and are also experimenting with genetic changes that could eliminate viruses like HIV. Even though viruses aren’t genetic diseases, certain gene edits have been shown to prevent the virus from spreading to new cells and to “destroy inactive HIV residing in the human genome by altering critical viral genes,” according to a look at genome surgery in MIT Tech Review. Experts even think these types of changes could eventually help treat complex conditions … Read more

CRISPR vs TALEN vs ZFN vs RNAi vs Homing Meganucleases

-Homing meganucleases are enormous and old-school
-ZFNs are compact (28:1 for input DNA:target) but obviously hard to target, so they may have specialized applications
-TALENs can be synthesized solid-phase, but they’re pretty huge (I think it was either 100 or 1k:1). The modularity is an improvement over ZFNs, which is why they were in fashion
-CRISPR are of course the best (1:1), although the extra machinery may be tough to deliver. Also, I’m curious as to the packing of the RNA–I feel like that might pose a problem for delivery? … Read more

CRISPR/Cas9 and Targeted Genome Editing

The development of efficient and reliable ways to make precise, targeted changes to the genome of living cells is a long-standing goal for biomedical researchers. Recently, a new tool based on a bacterial CRISPR-associated protein-9 nuclease (Cas9) from Streptococcus pyogenes has generated considerable excitement (1). This follows several attempts over the years to manipulate gene function, including homologous recombination (2) and RNA interference (RNAi) (3). RNAi, in particular, became a laboratory staple enabling inexpensive and high-throughput interrogation of gene function (4, 5), but it is hampered by providing only temporary Read more

CRISPR/Cas9 System for Targeted Genome Modification

The CRISPR/Cas9 system, Origene’s latest tool in genome editing, is a complete system for highly specific gene knockout with low cell toxicity. The CRISPR/Cas9 technology uses co-expression of a Cas9 protein with a guide RNA vector expressed from the human U6 polymerase III promoter. Cas9 unwinds the DNA duplex and cleaves both strands upon recognition of a target sequence by the guide RNA.

CRISPR/Cas9 Kit Features:
Complete kit for gene knockout using CRISPR
Two guide RNA vectors in for efficient cleavage
Knockin GFP-Puro for selection
pCas-Guide-scramble included as a negative … Read more

The CRISPR/Cas9 System & Genome editing

Clustered regularly interspaced short palindromic repeats (CRISPR) and a CRISPR associated protein (Cas9) comprise a system adapted from bacterial DNA known as CRISPR/Cas9. The CRISPR/Cas9 system act as an adaptive immune system allowing bacteria to recognize foreign DNA and cut it, destroying an invading virus or pathogen. CRISPR/Cas9 has been adapted to other organisms to make specific cuts in their genomic DNA.


CRISPR/Cas9 technology holds the potential to “edit” a patient’s genome, restoring genes with disease-causing mutations to their normal healthy sequence, deleting disease-causing genes, or improving genes … Read more